MOLECULAR GENETICS OF THE

HUMAN X

BY

DAVID ANDREW HARTLEY

a thesis submitted for the degree of

Doctor of Philosophy

in the

University of London

January, 1984 Department of Biochemistry St. Mary's Hospital Medical School London W2 IPG

1 Abstract

The comparison of frequency of recombination with

chromosomal position has been possible only in Drosophila melanogaster but it is known that chiasma distribution in

the human male (and many other species) is not random. A number of cloned DNA sequences have been isolated from a

library enriched for X chromosome DNA by flow cytometry.

The chromosomal loci complementary to these cloned

sequences have been investigated by hybridization to

somatic cell hybrid DNAs and by direct hybridization 1 in

situ' to human metaphase spreads. In this way a

cytological map of the X chromosome defined by DNA

sequence markers has been constructed. Genetic distances

complementary to the physical separations determined have

been estimated by determining the segregation of DNA

sequence variants at each locus in three generation

p e d i g r e e s .

The sequence variation is manifested as altered

restriction fragment migration in agarose gels. It is

intimated from these analyses that the pattern of

recombination frequency along the human X chromosome mirrors the non-random chiasma distribution seen along male autosomes. This supports the chiasmatype theory and has implications on the availability of genetic components

of the X chromosome to recombination.

2 To ray father in the hope of convincing him of life outside

o f E.coli

3 ...scientists getting their kicks when deadly disease can do as it pleases

results ain't hard to predict

Gil Scott-Heron

4 Ack n ov; ledgements

To my supervisor Bob Williamson for support. To my other supervisor Kay Davies for tolerance and PR. To

Graham Casey, Marion Stone, Teresa Davies and Paul Barton for teaching me the true meaning of cytogenetics. To Peter

Goodfellow for hybrid cells. To Paul North for microchip technology. To Helen Kingston and Dennis Drayna for

families and Lod scores.

To all the Biochemistry Department and in particular the 'X group' for tolerating my singing and my socks. To

Julian and Hark for endless attempts at the meaning of life and for going for it. To Jo, Sue, Teresa, /Auntie

Janet, Sissel, Pauline and Susie for demonstrating the superiority of two X . To Jack, Ali, Rob,

Graham, Tim, Conrad, Ola and Simon for coming a close s ec o n d .

To my family and friends.

To my skin and blister for never letting up in giving me a hard time (no more than I deserved).

To Jane.

5 Abbreviations dATP adenosine deoxyribonucleotide triphosphate AP RT adenosine phosphoribosyl transferase b p ba se pair 3 kb p 10 base pairs BSA bovine serum albumin o C degrees Centigrade u C i ,m C i ,Ci micro, milli, Curie dCTP cvtidine deoxyribonucleotide triphosphate DNA deoxyribonucleic acid DNAase deoxyribonuclease dpm disintegrations per minute E. coli Escherichia coli EDTA ethylene diamine tetraacetic acid G6 PD H glucose-6-phosphate dehydrogenase pg / ng , ug , mg , g pico, nano, micro, milli, gram dGTP guanosine deoxyribonucleotide triphosphate hrs hours HAT hypoxanthine adenine thymidine HGPRT hypoxanthine-guanosine phosphoribosyl transferase u l ,m l ,1 micro, milli, litre Lod logarithm of the odds nm,um,mm,cm nano, micro, milli, centimetre 2 cm squared centimetre mins minutes u M ,m M ,M micro, milli, molar cM centiMorgan PPO 2,5-diphenyloxazole PEG polyethylene glycol P probability RFLP restriction fragment length polymorphism RFLV restriction fragment length variant rpm revolutions per minute RNA ribonucleic acid RNAase ribonuclease RPMI-1640 Roswell Park Memorial Institute-1640 m e d i u m SSC saline sodium citrate secs seconds SDS sodium dodecyl sulphate SD standard deviation Tris 2-amino-2(hydroxymethyl)-propane-1,3-diol dTTP thymidine deoxyribonucleotide triphosphate UV ultraviolet V volts v / v volume per volume of water w / V weight per volume of water

6 F i g u r e s ge

Secondary screenings of X-enriched library recombinants with human, Horl9X and mouse DMAs. 60

Screenings of X-enriched library with Horl9X and

MCP-6 DMAs. 62

EcoRI digests of recombinants DX1-12. 64

Recombinants DX1-12 hybridized to human and lambda DMAs. 66

EcoRI digests of human, Horl9X, IV7I-5, MCP-6 and mouse DMAs. 71

Somatic cell hybrid DMAs hybridized to DX24 and

RD6 . 73

Somatic cell hybrid DMAs hybridized to S21 and

DX4 . 75

Somatic cell hybrid DMAs hybridized to DX7 and

DX6 . 76

4C11 hybridized to a metaphase spread. 82

Distribution of grains over chromsome 6s after

'in situ' hybridization to 4C11. 85

Somatic cell hybrid DMAs hybridized to 4C11. 86

Repetitive seguences hybridized 'in situ' to metaphase spreads. 83

RC8 hybridized to a metaphase spread. 90

Distribution of grains over all chromosomes after

'in situ' hybridization to RC8. 91

7 15. Distribution of grains over the X chromosomes

after 'in situ' hybridization to RC8. 93

16. LI.28 hybridized to a metaphase spread. 95

17. Distribution of grains over all chromosomes after

'in situ' hybridization to LI.28. 96

18. 52A hybridized to a metaphase spread. 99

19. Distribution of grains over all chromosomes after

hybridization to 52A. 100

20. S21 hybridized to a metaphase spread. 102

21. Distribution of grains over all chromosomes after

hybridization to S21. 103

22. DP31 hybridized to a metaphase spread. 106

23. Distribution of grains over all chromosomes after

hybridization to DP31. 107

24. DX13 hybridized to a metaphase spread. 109

25. Distribution of grains over all chromosomes after

hybridization to DX13. 110

26. Distribution of grains over the X chromosome

after hybridization to RC8, LI.28, DP31, S21, 52A

and DX13. 113

27. Cvtological map of the X chromosome. 114

28. The TaqI polymorphism detected by RC8. 117

29. The TaqI polymorphism detected by S21. 119

30. The TaqI variants detected by LI.28 and DP31 in

the pedigree D3. 123

31. Pedigree D3. 124

32. Pedigree D2. 126

8 33. The TaqI and Bglll variants detected by DP31 and

DX13 in the pedigree BD26. 128

34. Pedigree BD26. 129

35. The physical and genetic map of the human X

chromosome. 133

9 Tables

Page

1. Recombinants picked from the X-enriched library -

insert sizes and hybridization to total human

DliA. 69

2. Hybridization of recombinants to somatic cell

hybrids - chromosomal localization. 78

3. Statistical analysis of 4C11 'in situ'

hybridization. 84

4. Statistical analysis of RC8 'in situ'

hybridization. 92

5. Statistical analysis of LI.28 'in situ'

hybridization. 97

6. Statistical analysis of 52A 'in situ'

hybridization. 101

7. Statistical analysis of S21 'in situ'

hybridization. 104

8. Statistical analysis of DP31 'in situ'

hybridization. 108

9. Statistical analysis of DX13 'in situ'

hybridization. Ill

10. Restriction fragment variants in eight mothers

detected by RC8, LI .28,DP31, S21, 52A and DX13. 121

11. Lod scores at different values of recombination

fraction between the loci detected by LI.28 and

DP31 and between RC8and Ll.28. 131

1 0 Contents Page

Abstract 2

Dedication 3

Quotation 4

Acknowledgements 5

Abbreviations 6

List of Figures 7

List of Tables 10

1 . Introduction 14

1 .1 . The eukaryotic chromosome 15

1 .2. Meiosis and the reassortment of 20

1.3. Recombination between linked genes and the

construction of linkage maps 22

1.4. Crossing over and chiasmata 25

1 .5 . Localized recombination 27

1 .6 . Cytological and genetic markers inman 30

1 .7 . Flow cytometry and molecular cloning of the

human X chromosome 33

1.3 . Summary and aims 35

2. Materials and Methods 37

2.1. Materials 38

2.2. Bacterial and phage strains 41

11 2.3. General methods 41

2.3.1. Restriction enzyme digestion 41

2.3.2. Ethanol precipitation of nucleic acids 42

2.3.3. Agarose gel electrophoresis 43

2.3.4. Autoradiography 43

2.3.5. Gel filtration 43

2.3.6. Determination of radioactivity 44

2.4. Specific methods 44

2.4.1. Preparing high molecular weight DHA from

tissue culture cells 44

2.4.2. Small scale preparation of bacteriophage DMA 45

2.4.3. Large scale preparation of bacteriophage DNA 46

2.4.4. Labelling DMA by nick translation 47

2.4.5. Screening lambda recombinants 43

2.4.6. Southern transfers 50

2.4.7. Competing repetitive sequences in Southern

blot hybridizations 51

2.4.8. Preparation of metaphase spreads 51

2.4.9. G-banding of metaphase chromosomes 52

2.4.10. 'In situ' hybridization 53

2.4.11. Scoring hybridized spreads 55

2.4.12. Pedigree analysis 56

12 3. Results 57

3.1. Screening recombinant bacteriophage 'in situ' 53 3.2. Isolation and characterization of individual

recombinant bacteriophage S3 3.3. Chromosomal localization I. Somatic cell

hybrids S3 3.4. Chromosomal localization II. 'in situ'

hybridization 79 A. Introduction 79

B. Cytological mapping of the X chromosome 29 C. Summary of 'in situ' hybridization data 112 3.5. Restriction fragment length polymorphisms 115

A. RFLP and heterozygote detection 115 B. Inheritance studies 122 3.6. Genetic distances - a genetic map of the X 130

4. Discussion 135

4.1. DBA sequence organization of the human X chromosome 136

4.2. The construction of chromosome maps in

r1 amma 1s 133 4.3. Localized recombination 143

5 . Summary and perspectives 154

6 • References 15 3 hrrata 16 6

13 C H AP TE R 1

Introduction

14 1.1. The Eukaryotic chromosome

The chromosome, first observed by Waldeyer in 1888, can be defined as a linkage structure consisting of a specific linear sequence of genetic information. The eukaryotic chromosome has two main biological functions.

The first is the transmission of genetic information from cell to cell and generation to generation and the second the ordered release of this information to control cellular function and development. The chromosomes of higher eukaryotes are characterized by their complex structure and by their behaviour during mitosis and meiosis when they undergo extraordinary changes in morphology.

The dissection of the chromosome by microscopic and biochemical techniques has revealed much of its molecular architecture. By thin section techniques and surface spreading of lysed cells the electron microscope reveals

'thick' 20-30nm fibres which unfold at low ionic strength to the characteristic 'beads on a string' structure

(DuPraw, 1970). This beaded structure corresponds to the now well established nucleosome which consists of two molecules each of the histones H2A, H2B, H3 and H4 with

140-200 base pairs of DMA (Oudet et al., 1975). The 20-

30nm fibre is interpreted variously as a solenoid of nucleosomes (Finch and Klug, 1976) or a repeating unit of

6-10 nucleosomes as condensed 'superbeads' (Hozier et al.,

1977).

15 A series of experiments have addressed themselves to the question as to whether the DNA is continuous and unineme in each chromosome. Observations on the deoxyribonuclease digestion of lampbrush chromosomes

(Gall, 1963), on the isotopic labelling of replicating chromosomes demonstrating semi-conservative inheritance of labelled DNA (Taylor, 1963), on the bromodeoxyuridine label distribution following sister chromatid exchange

(Latt, 1981) and on the viscoelastic properties of large

Drosophila melanogaster DNA molecules (Kavenoff and Zimm,

1973) are most simply explained by the conclusion that there is one uninterrupted DNA duplex per chromosome.

Interphase chromatin is visualized in the electron microscope as twisted 20-30nm fibres. At mitosis these fibres condense to yield the characteristic distinguishable structures of metaphase chromosomes.

Models to explain the compaction of the 20-30nm fibre have included further helical packing in a 'supersolenoid' (Bak et al., 1977) and a 'folded fibre' arrangement of repeated transverse and longitudinal folding of the 20-30nm fibre

(DuPraw, 1968; Comings, 1972). The organization of metaphase chromatin around a central backbone or core was first proposed by Taylor in 1958. Elegant work from the laboratory of Laemmli has confirmed this model. By competing out the histones using polyanions Laemmli and his coworkers have demonstrated that a scaffold is retained with loops of DNA emanating outwards, each loop

16 varying in length from 15-30um or approximately 45-90kbp

(Paulson and Laemmli, 1977). They have proposed the model that the histones coil up the DMA into the 20-30nm solenoid fibres which are attached to a non-histone protein scaffold (Laemmli et al., 1977). Using different procedures to isolate metaphase chromosomes the packing of the DNA loops is revealed. Electron microscopy of chromosomes isolated in the presence of divalent cations reveals the lOnm DMA duplex and in the presence of ImM

MgCl^ reveals the 20-30nm nucleosome solenoid. Chromosomes extracted in hexylene glycol reveal 50-60nm knobs and retain the dimensions of the intact metaphase chromosome.

These knobs are interpreted as supercoiled solenoids

(Marsden and Laemmli, 1979).

The determination of the metaphase chromosome structure, dividing the DNA into loops or domains directly reflects the structure of the lampbrush chromosome of meiotic cells (Miller and Hamkalo, 1972).

The proposal that interphase chromatin is also maintained in domains is strengthened by nuclease digestion studies of interphase nuclei (Igo-Kemenes and Zachau, 1977).

Centrifugation studies of lysed Drosophila melanogaster cells revealed supercoiled circular DNA structures of approximately 85kbp (Benyajati and Uorcel, 1976) and electron microscopic observations of mouse interphase nuclei suggest a non-histone protein skeleton with approximately 54kbp loops of DNA emanating outwards (Igo-

Kemenes et al., 1982).

17 Purification and reassociation analysis of human genomic DMA has led to our understanding of the DMA sequence arrangement in the chromosome. Human DMA sequences may be broadly classified into three classes

(Jelinek and Schmid, 1982). The first is the non- repetitive fraction present in less than thirty copies of each sequence and consisting of the majority of the coding and intervening sequences. The second type are the moderately repetitive sequences present in 50-1000 copies consisting of long and short sequences, some of which may be coding, interspersed with unique DMA. The third class are the highly repetitive sequences present in greater 4 than 10 copies and consisting of fairly short sequences in clusters. Dispersed repetitive sequences may be further divided into families of repeats which are often defined by common restriction sites. These type of sequences make up 15-30% of the genome by mass (Jelinek and Schmid, 1982) and their paradigm is the so-called Alu family which alone accounts for 3-6% of the genome (Houck et al.,

1979). Alu sequences have been found both between and within genes (Jelinek and Schmid, 1982). Other examples of dispersed repeats include the Hindlll repeat (Manuelidis and Biro, 1982), the 6.4kbp repeat found in the beta- globin cluster (Jelinek and Schmid, 1982), the Kpnl repeat

(Shafit-Zagardo et al., 1982) and the genes encoding the small nuclear RNAs (Jelinek and Schmid, 1982). Repetitive

DMA sequences are also found clustered, particularly the

18 highly repetitive sequences which make up the bulk of the heterochromatin found predominantly in pericentric and telomeric locations (Brutlag, 1980). Moderately repetitive sequences may also be clustered and examples include the so-called ocRI repeat (Darling et al., 1982) and the 2kbp

BamHI repeat (Yang et al., 1982; Willard et al., 1982).

In terms of function, repetitive DNA sequences remain an enigma. It has been proposed that they are pu rely parasitic and any function is incidental to their maintenance in the genome (Doolittle and Sapienza, 1980;

Orgel and Crick, 1980). An alternative hypothesis is that they regulate transcription or the processing of primary transcription products to mature messenger RNA molecules

(Davidson and Britten, 1973). An attractive possibility is that they organize the DNA into the domains seen by electron microscopy and biochemical techniques. This hypothesis is supported by the observation that repetitive

DNA is enriched in the insoluble fraction after nuclease digestion of interphase nuclei (Jeppersen and Bankier,

1979) .

Since the domain structure of chromosomes is apparently conserved throughout the cell cycle and the DNA sequence also exhibits an interspersed pattern, this has provoked the hypothesis that these domains are functional units. They may be units of replication (Hand, 1978) and/or transcription (Stadler et al., 1980). Indeed, with recent interest in the role that different configurations the DNA helix might adopt in distinguishing active and

19 *

inactive chromatin, a model for DMA domain activation has

been proposed (Rich, 1982). In this model the DMA loops

out in segments from a nuclear matrix/scaffold protein

which isolates the DMA for supercoiling between the

different domains. Individual domains are seen as inactive

when a key control region takes the so-called Z-DNA

configuration but become activated when they return to the

normal Watson-Crick B-DNA configuration. This activation

would be associated v/ith a change in superhelical density

leading to a non-random uncoiling and increased

availability of genes located in the domain to polymerases

and/or protein effectors.

1.2. Meiosis and the reassortment of genes

The evolutionary success of sexually reproducing

organisms is evident even if the selective forces

responsible for the origin and maintenance of eukaryotic

sex are not (Maynard-Smith, 1978). Oscar Hertwig first

discovered in 1875 that fertilization, the basis of sexual

reproduction, concerns the nucleus. Waldeyer's subsequent

cytological discovery of the chromosomes revealed that

they too were involved in fertilization. Our understanding

of the second essential characteristic of eukaryotic sex,

meiosis, springs from two suggestions made by Weismann

(Weismann, 1887). The first was that meiosis would be

2 0 found to compensate for fertilization in the life cycles of both sexes and all organisms. The other, added with the purpose to make a clean separation between Darwin and

Lamarck, was that the development of sexual reproduction in evolution depends on the value of meiosis in exposing the results of genetic recombination to natural selection.

The implications of this statement have led to the development of much of what are today the sciences of modern genetics and evolutionary biology.

The behaviour of the chromosomes during meiosis has been characterized by microscopic analysis. The four copies of each chromosome are distributed to different progeny cells by successive divisions. Preceding the first division is a lengthy prophase which, in some organisms, can last several months. In leptotene at the beginning of prophase, the chromosomes are seen as very fine extended threads which approach each other during zygotene and begin to pair side by side. At the end of leptotene, lateral components have been formed in the interval between two chromatids of unpaired chromosomes

(Westergaard and von Wettstein, 1972) and subsequently as partner chromosomes synapse in zygotene, starting at the telomeres (Callan and Perry, 1977), the lateral components associate with a central core to form the synaptonemal complex (Moses, 1958). Synapsis is complete by the stage of pachytene. The double nature of the synaptonemal complex is clearly revealed at diplotene when the chromosomes separate along much of their length except at

2 1 certain restricted points termed chiasmata. These

chiasmata are thought to mark the site at which genetic

exchange has taken place (Janssens, 1909). From

experimental alterations of cross over frequencies and

biochemical studies it has traditionally been held that

synapsis must be complete before exchange can occur. This

viewpoint has not remained unchallenged however (Maguire,

1977). The final stage of meiotic prophase is diakinesis

when the bivalents coil up to form shorter and thicker

chromosomes and the chiasmata have a tendency to move

towards the ends of the the paired homologues. This

movement is more pronounced in shorter chromosomes and may

be due to torsional strain induced by chromosome

condensation. Chiasma terrninalization may be complete,

partial or entirely lacking (Darlington, 1931).

The spindle is organized in prometaphase I and

pairing configurations line up on the equatorial plate in

metaphase I, to be distributed to opposite poles in

anaphase I. The second meiotic division which is

mechanically similar to mitosis then proceeds, a membrane

surrounding the four haploid nuclei at telophase II.

1.3. Recombination between linked genes and the

construction of linkage maps

The segregation and transmission of chromosomes at

22

» meiosis provided Sutton and Boveri with a physical manifestation of Mendelian inheritance (Sutton, 1903).

VJith the discovery of an increasing number of genes in

Drosophila melanogaster came the realization that some of these genes must reside on the same chromosome. When these genes were studied in nonallelic pairs or groups, they yielded ratios that frequently departed from random assortment. This led to the definition by Morgan of linkage or synteny in 1911 (Morgan, 1911) and of crossing over in 1912 (Morgan and Cattell, 1912). The proposition was that genes are arranged in a linear order, each having a fixed position on the chromosome. Sturtevant subsequently proposed that the extent of recombination between two genes was a measure of their distance apart on the chromosome and that recombination 'distances' were additive (Sturtevant, 1913). The unit distance corresponds to 1% recombination frequency and is often referred to as the centiMorgan (cM).

The pioneering work of Morgan, Muller, Bridges and

Sturtevant has led to the accumulation of genetic data on the frequencies of crossing over, thus enabling the construction of genetic or chromosome maps in which the serial order of the genes and their genetic spacing have been accurately determined.

The principles governing gene order, linkage and crossing over are undoubtedly applicable to analysing inheritance of Mendelian traits in humans. However, human families are small, the lifetime of the experimenter is

23 that of the subject and genetic manipulation is prohibited

on ethical grounds. Consequently a likelihood test for

linkage has been devised by Morton and is referred to as

the sequential or lods method (Morton, 1956). This yields

likelihood ratios as to whether the data obtained from any

particular pedigree are the consequence of recombination

occurring at a certain frequency (theta) or the

consequence of independent assortment. By giving theta in

the numerator of the equation a series of values from 0 to

50 and by keeping theta in the denominator at 50 ( a value

indicating no linkage), each pedigree can be analysed and

a series of likelihood ratios obtained. By making use of

the logarithm of the odds (Lod), the likelihood ratios

from different pedigrees can be summed and the

recombination frequency giving the highest lod value

determined. This value for

Lod=log^Q x likelihood of observed pedigree assuming 0=0 likelihood of observed pedigree assuming 0=50

theta is known as the maximum likelihood estimate and a

lod value of +3.0 is very strong evidence of linkage whilst a value of -2.0 is equally strong evidence of

independent assortment. Any value between would provide

only an equivocal estimate.

24 1.4. Crossing over and chiasmata

The proposal that the chromosomal basis of crossing

over lay in the chiasmata observed at diplotene originated

with Janssens in 1909. Stern, using Drosophila and

Creighton and McClintock in maize provided experimental

evidence that crossing over involved a physical exchange of chromatin by using homologues that could be

distinguished cytologically by the presence of

translocations (Stern, 1931; Creighton and McClintock,

1931). The first direct physical evidence in favour of Janssens' chiasmatype theory was provided by 3 autoradiographic analysis of H- thymidine labelled

Romalea microptera rneiotic chromosomes (Taylor, 1965) and

subsequently using BUdR labelled chromosomes in Locusta

migratoria (Tease and Jones, 1973). Chiasma frequencies have been used to estimate genetic distances in those organisms whose genetic map is inadequate. To what extent this is justified can be

determined from attempts to quantitatively correlate

chiasma frequencies and cross over frequencies. In the

lily, the exhaustive study of Brown and Zohary has

demonstrated a 1:1 correspondence of chiasmata to

chromosome exchange (Brown and Zohary, 1955). In maize, a comparison of the total map lengths of the known linkage groups to average chiasma frequencies approaches 1:1

correspondence as the chromosomes become more accurately

mapped (Neuffer and Coe, 1976). Observations on chiasma

25 i frequencies in human male spermatocytes (Hulten, 1974) have led to the estimation of 30 Morgans for the total map length of the (McKusick, 1980).

Comparisons of genetic maps based on recombination and chiasma frequencies betv/een species reveals a decrease in recombination frequency per unit DNA with increasing genome size. It is tempting to speculate that this reflects a constant level of recombination per gene as the amount of DNA per gene appears to increase with increasing genome size.

Certainly it is apparent that the number of chiasma is under genetic control and may be selected for (Shaw,

1974). Studies on recombination in meiotic mutants of lower eukaryotes, Drosophila and plants (Baker et al.,

1976) support this observation. The intrinsic factors affecting the frequency of crossing over appear to be sex, age and genotype. Thus the frequency of crossing over may be identical between the two sexes (or in the anthers and ovaries of dioecious plants), as in the garden pea; higher in females than in males, as in the mouse, man and rat; or higher in males than in females as in the pigeon. Extreme sexual divergence in regard to frequency of exchange is also known. In male Drosophila melanogaster there is no crossing over and no synaptonemal complex formation, whilst the female silkmoth, Bombyx mori experiences no crossing over at meiosis (White, 1973). Where linkage differentials exist between the sexes and where adequate

26 data are available, it is the heterogametic sex in which crossing over is either lowered in frequency or abolished.

1.5. Localized recombination

The first demonstration that recombination does not occur randomly came from early studies in Drosophila which indicated that one cross over has a tendency to interfere with the occurrence of another in its vicinity (Muller,

1916). This corresponds to early analysis of the distribution of chiasmata along the chromosome demonstrating a significant departure from the Poisson distribution expected for random location (Haldane, 1931).

Mather first proposed that the location of the first chiasma to form on any one chromosome is relatively fixed whilst the second (i f any) is determined by an interference exhibited by the first (Mather, 1938) and work on locust chromosomes is consistent with this hypothesis (Fox, 1973).

There are many examples of localized chiasmata in nature. Virtually all chiasmata in the male meiosis of the grasshopper Stethophyma grossum occupy a procentric location whilst in the female greater than 94% of the chiasmata are located interstitially or distally. In the palmate newt Triturus helveticus male chiasmata have a proterrninal localization but in the female they are all intercalary. The opposite occurs in the Great Crested newt

27 Triturus cristatus where the chiasmata in the female are procentric in contrast to the liberal distribution found in the male (Callan and Perry, 1977).

The chiasma distribution for the human male has been described (Hulten, 1974) and it appears that chiasmata occur more frequently at a proterminal location (Laurie et al., 1982) and that interference extends for 3.5% of the total autosomal length although not at all across the centromere. Variation in crossing over frequency at different cytological locations has been well characterized. The classic example is the reduction of crossing over seen in the vicinity of the centromeres of Drosophila melanogaster. Translocations or inversions bringing euchromatic material adjacent to the heterochromatic centromeric regions bring about a reduction in crossing over frequency in that euchromatin. In addition, proximal markers can be induced to recombine more freely when translocated away from their procentric location (Schalet and Lefevre, 1976). Other local differences in crossover frequencies have been found. For example, in the distal portion of the left arm of the X chromosome in Drosophila the genes y and w are separated by 75-80 bands and recombine at a frequency of 1.5%. The same recombinational frequency is found between the genes w and _fa yet these are separated by only 4-5 bands. However, it is not clear whether the haploid DNA content per band is relatively

28 constant in Drosophila (Lefevre, 1971).

The study of meiotic mutants provides additional

evidence for the differing affinities of different regions

of the chromosome for recombination (Baker et al.# 1976).

The mutation rec-1 in Neurospora crassa causes a 10-25

fold increase in heteroalleiic recombination of the his-1

and nif-2 loci without any affect on 19 other markers. In

Drosophila/ a group of mutants (mei-41, mei-218, mei-251, mei-S282, mei-B, abo, mei-68^1^) decrease exchange most

severely in regions where crossing over per unit length is

relatively high in the wild type (distal intercalary

positions) and less severely where it is relatively low

(procentric and telomeric positions). A mutant has also been described that governs only the distribution and not

the overall frequency of recombination, mei-352, which

seems to allow a more random distribution of exchanges.

It is clear that a precise determination of the

position and frequency of crossing over in relation to the chromosome is facilitated by the genetic and chromosome maps available in Drosophila. In no other eukaryote has

such a comparison been possible. The extensive genetic map of the mouse has permitted the conclusion to be drawn that

the known genes do not follow the Poisson distribution

expected if they are evenly distributed in the genome and chiasmata form randomly (Lyon, 1976). However, it remains

unclear whether the discrepancy lies with the distribution of genes or chiasmata or both.

29 1.6. Cytological and genetic markers in man

In recent years two significant advances have changed the face of mammalian gene mapping. The first is the development of somatic cell genetics permitting the

isolation of chromosomes and the second the introduction of recombinant DMA techniques allowing the purification of

specific RNA and DMA sequences which can detect homologous genomic loci.

Production of interspecific cell hybrids can be achieved at will using inactivated Sendai virus or polyethylene glycol to mediate cell fusion (Ringertz and

Savage, 1976). Subsequent growth of human x rodent hybrids results in a continual disappearance of human chromosomes

(VJeiss and Green, 1967). However, specific human chromosomes will be retained if that chromosome carries a selectable marker in an appropriate rodent background.

Thus human x mouse hybrids can be compelled to retain the gene for hypoxanthine-guanosine phosphoribosyl transferase

(lIGPRT) by growth on HAT (hypoxanthine, aminopterin, thymidine) medium. This enzyme activity segregates with human glucose-6-phosphate dehydrogenase (G6PDH) activity and the presence of the human X chromosome (Nabholz et al., 1969) and thus facilitates the assignment of any human genes expressed in such hybrids to the X chromosome.

The use of human parent cells containing derivative X chromosomes such as translocations and deletions generates

HAT resistant hybrids containing differing amounts of X

30 chromosomal material (Ricciuti and Ruddle, 1973). This permits the intra-chromosomal localization of X-linked genes expressed in the hybrid.

The isolation of restriction enzymes which cleave at specific DNA sequences and ligases and polymerases which can link fragments together has facilitated the construction of hybrid or chimeric DNA molecules (Jackson et al., 1972). In this way, hybrid plasmid or bacteriophage DNA molecules can be obtained in large amounts by growth in bacteria and specific DNA sequences may be purified. For example, a 'library' of cloned sequences of the human genome can be constructed by linking human DNA, partially digested with a restriction enzyme, to bacteriophage DNA and growing the resultant recombinant phage in bacteria (Maniatis et al., 1978).

Many techniques now exist for purifying individual DNA or

RNA segments or molecules which code for a known product

(Maniatis et al., 1982). Cytological localization of genes is greatly facilitated by the availability of cloned DNA due to its ability to reassociate with its complementary copy in the human chromosome in a sequence specific fashion. A method for detecting specific sequences in the human genome has been devised by Southern (1975). Using this method with genomic DNA from a human x rodent somatic cell hybrid, the cytological location of the DNA complementary to the cloned probe fragment can be determined.

31 An alternative and powerful approach to the use of recombinant DMA probes in conjunction with somatic cell hybrids is the technique of 'in situ' hybridization (Gall and Pardue, 1969). /applicable only to the cytological localization of reiterated sequences initially, the technique has been refined for the location of DNA sequences present in a single copy in the human genome

(Harper and Saunders, 1981). Radioactively labelled DNA probes are used to reassociate with the chromosomal DNA of human metaphase spreads fixed to glass slides and hybrids detected by autoradiography. In this fashion, the chromosomal locus for a number of genes has been determined (Harper et al., 1982; Malcolm et al. , 1982;

Trent et al., 1982). This technique offers the advantage of a single high resolution localization, often to within a single chromosome band.

It is manifest that cloned DNA fragments can detect cytological loci present in a single copy in the genome.

The same fragments may also detect genetic loci as they can demonstrate DNA sequence variants on different chromosomes which will be inherited in a Mendelian fashion. Such variants are detected most readily as restriction digest fragments whose mobility in agarose gels is altered. This restriction fragment length polymorphism (RFLP) is generated by mutation leading to the loss or gain of restriction sites or insertion or deletion of DNA between two sites. It has been proposed that such RFLP loci can be used to generate linkage maps

32 of whole chromosomes and eventually the entire human genome (Botstein et al., 1930; Davies, 1981). Such chromosome linkage maps can be compared to cytological maps defined by the same DMA loci. In this way, the position and frequency of crossing over can be precisely determined in a fashion analogous to the Drosophila genetics of the first half of this century.

1.7. Flow cytometry and molecular cloning of the

human X chromosome

The human chromosome most amenable to linkage analysis is the X due to the hemizygous nature of the male and subsequent absence of detectable crossing over. The odds that any DNA fragment (from a 'library' of similar sized fragments) is derived from the X are approximately

18:1. This makes the construction of an X chromosome map from a total genomic DNA library impractical. The two most powerful approaches of making DNA libraries enriched for a particular chromosome are the use of interspecific somatic cell hybrids and the use of flow cytometry and flow sorting.

Isolating DNA sequences specific to a human chromosome from a somatic cell hybrid has been used for chromosome 11 (Gusella et al., 1980). The technique relies on the dispersed nature of most repetitive DNA sequences and their species specificity. Human DNA sequences in the

33 human x Chinese hamster hybrid were identified by reacting individual recombinants from a genomic library with radiolabelled total human DNA. Only the repetitive DNA sequences are in sufficient concentration in the probe to allow autoradiographic detection. Human single-copy DNA sequences found on the same recombinant molecule were isolated and used as a probe for intra-chromosomal localization.

Flow cytometry is a recent approach to cytogenetics that enables separation of metaphase chromosomes on the basis of their reaction with an appropriate flourochrome.

Chromosomes are stained in aqueous suspension and constrained to flow at high speed through a narrow laser beam that excites the stain. The photometrically measured emitted flourescence permits the human chromosomes to be sorted into twelve main groups (Carrano et al., 1979). The molecular cloning of DNA extracted from sorted chromosomes facilitates the construction of recombinant 'libraries' greatly enriched for particular chromosomal DNA sequences.

A chromosome preparation enriched for the X by flow sorting had its DNA extracted and digested to completion with the restriction endonuclease EcoRI. Fragments were ligated to bacteriophage lambda DNA and individual recombinant molecules packaged into infectious viral particles 'in vitro' (Davies et al., 1981). The 50,000 recombinants thus obtained provide a starting point for the construction of a cytogenetic map of the human X chromosome.

34 1.8. Summary and aims

The human chromosome contains a single, continuous

longitudinally arranged DDA molecule. This molecule

appears to be organized in domains with 'loops' of DtIA constrained to a non-histone protein matrix seen as a

scaffold structure at metaphase. Repetitive DNA sequences might play a role in the organization of such a structure, may be involved in gene regulation or indeed have no function other than self maintenance. The domain structure of the chromosome may be crucial to its function in the way genes are replicated, transcribed and recombine at m e i o s i s .

Little is known of the relation of the chromosome to meiotic recombination as genetic maps are extensive in only a few eukaryotic organisms, principally Drosophila melanogaster. It seems clear that the cytologically observed chiasmata are the sites of genetic exchange and evidence for non-random positioning of chiasmata is abundant; their frequency and positioning appears to depend on sex, age and genotype. Evidence for localized recombination is present only for Drosophila whose salivary chromosome bands have been correlated to transcription units. Recombination in the human can be investigated using cloned DNA sequences. These can detect chromosomal loci and reveal inheritable sequence variants.

A genomic library of 50,000 recombinants greatly enriched for the human X chromosome DMA provides the

35 starting material for this study. The cytological location

of individual DNA fragments is determined by the use of

interspecific somatic cell hybrids and 'in situ' hybridization. Genetic distances are estimated by

following the inheritance of variant restriction fragments

in pedigrees and summing information by the sequential or

lods method. In this way the frequency of crossing over can be determined and compared in different parts of the chromosome.

3G CH AP TE R 2

Materials and Methods

37 2.1. Materials

Ch em ic al s

With the following exceptions, all chemicals were

Analar grade supplied by British Drug Houses (BDH), Poole,

England. Sodium chloride, Trizma base, dextran sulphate, ethidium bromide, orange G and bromophenol blue were from the Sigma Chemical Co., St. Louis, Missouri, U.S.A.

Spermidine trihydrochloride was from Fluka AG, Buchs,

Switzerland. Ficoll and polyvinyl pyrrolidone were from

Pharmacia, Uppsala, Sweden.

Isotopes

All radioisotopes were supplied by Amersham

International, Amersham, England.

Chromatography, electrophoresis, transfer, and

hybridization

Sephadex was from Pharmacia. Polymer wool was from

Interpet, Dorking, Surrey. Agarose was supplied by Miles

Laboratories Inc., Kanakee, Illinois, U.S.A. or by

Bethesda Research laboratories (BRL), Rockville, Maryland,

U.S.A. Nitrocellulose filters were from Schleicher and

Schull, Dassel, West Germany. Bovine serum albumin (BSA) was from Sigma.

Photography and autoradiography

PanF film, Phenisol and ID-II developers, llypam fixer

38 and K2 and L4 nuclear track emulsions were from Ilford,

Basildon, England. Polaroid film came from Polaroid (UK)

Ltd., St. Albans, England. RX medical X-ray film and Each

II intensifying screens were from the Fuji Photo Co. Ltd.,

Tokyo, Japan. The UV light source was a short wave transilluminator from UV Products Inc., San Gabriel,

California, U.S.A. NTB-2 nuclear track emulsion and X-OMAT

XAR-5 film was from Eastman Kodak, Rochester, New York,

U.S.A.

Enzymes

EcoRI, Hindlll, TaqI, Bglll, SstI and BamHI were from

BRL. Additional EcoRI was a kind gift from Dr. L. VJoodhead in this laboratory. DNAase I, E.coli DNA polymerase I and proteinase K were from Boehringer Corp., Mannheim, West

Germany. RNAase A was from Sigma. Nick translation kits were supplied by Arnersham.

Nucleic acids

Herring sperm DNA and polyadenvlic acid were from

Sigma Chemical Co. Deoxyribonucleotides were from

Boehringer. Bacteriophage lambda DNA was from BRL.

Bacteriological reagents

Agar v/as from Difco Laboratories, Detroit, Michigan,

U.S.A. Yeast extract and tryptone were supplied by Oxoid

Ltd., Basingstoke, England. Sterile 90mm petri-dishes were

39 from Sterilin Ltd., Teddington, England. Bio-assay dishes were from Nunc, Roskilde, Denmark.

Tissue culture reagents

RPMI-1640 medium, foetal and new born calf serum and non-essential amino acids were from Gibco Europe Ltd.,

Uxbridge, England. Glutamine, kanamycin, colchicine and heparin were from Sigma. Phytohaemagglutinin was from

Wellcome Diagnostics, Dartford, England. Tissue culture grade sterile plastic flasks were from Sterilin Products.

Cytogenetic reagents

Lipsol detergent was from Lip (Equipment and

Services) Ltd., Shipley, England. Phosphate buffer tablets, Giemsa and Leishmann's stain were from BDH.

Microscope slides and coverslips were from Chance Propper

Ltd., Warley, England and Holdtite rubber adhesive was purchased from F.W. Woolworths Ltd., England.

Microscopy

All brightfield optics used a Reichert Polyvar equiped with a lOx eyepiece, lOOx objective and automatic camera all from C. Reichart, Vienna, Austria.

40 2.2. Bacterial and phage strains

LE392 and LE831 are derivatives of ED803 (hood,

1966). Their genotypes are:- — + + LE392 : rk mk recA supE supF gal met — + — LE831 : rk mk recA supE supF gal met

Plasmid recombinants were propagated on strains

HB101, (Boyer and Roulland-Dussoix, 1969) or MC1061

(Casadaban and Cohen, 1980).

Their genotypes are:-

HB101 : s t rr rB mB recA pro gal r _ + + M C I 061 : str rB mB recA ara leu

All bacteriophage were human recombinants in the vector XgtV/ES. "Xb (Tiemeier et al., 1976) v/hose genotype

is

"X nin5 att-red Wam403 EamllOO SamlOO

Plasmid recombinants LI.23 (Davies et al., 1983), 52A

(Drayna et al., 1984) and DP31 (Page et al., 1982) were all in pBR322 .

2.3. General methods

2.3.1. Restriction enzyme digestion

Digests were set up with the appropriate amount of

DMA, a tenfold concentrate of buffer specified by the manufacturer, 4mH spermidine trihydrochloride, 100 ug/ml nuclease-free BSA, enzyme and double distilled water to

41 bring the DMA concentration to between 0.1 and 0.2mg/ml.

For bacteriophage DMA, 1 unit of enzyme was added per ug

of DMA whilst for human, rodent or somatic cell hybrid

DMA, 2.5 units per ug were added. Digests were performed

in 1.5ml snap-shut eppendorf tubes at the temperature

specified by the supplier. After an incubation time of 1-

2hrs per ug of DMA the digest was placed on ice and a

small sample electrophoresed on an agarose gel to

determine the extent of reaction. Completed digests were

electrophoresed immediately or following phenol and

chloroform/isoamyl alcohol extraction, ethanol

precipitated and re-suspended in 10mM Tris-HCl pH7.5, ImM

EDTA, (TE buffer), at a concentration of 0.4mg/ml.

2.3.2. Ethanol precipitation of nucleic acids

DMA was routinely precipitated by the addition of 0.1 volume 3H sodium acetate plus 2.5 volumes of ethanol.

Bacteriophage and plasmid DMAs were chilled for 2hrs- overnight at -20°C and pelleted by centrifugation for lOmins in an eppendorf inicrofuge (Eppendorf 5412). The supernatant was removed and the pellet washed in 70% ethanol. After re-spinning, the supernatant was removed and the pellet dried under vacuum. High molecular weight

DMAs were lifted directly with a closed pasteur pipette and rinsed in 70% ethanol. The precipitate was allowed to dry in air before redissolving in an appropriate volume of

TE buffer, as were dried pellets.

42 2.3.3. A-garose gel electrophoresis

Agarose gels for molecular weight analysis and/or

Southern transfer were electrophoresed in E-buffer (40mM

Tris, 20mM sodium acetate, 2mM EDTA, pll7.7) containing

0.5ug/ml ethidium bromide. Gels were made by dissolving

the appropriate weight of agarose powder in E-buffer and

0.5ug/ml ethidium bromide and boiling the solution. The

solution v/as allowed to cool before pouring into a gel

mould. After the gel had set, the slot formers were

removed. Samples were loaded in 2% w/v Ficoll plus an

appropriate amount of orange G as a tracking dye.

Electrophoresis v/as carried out in an electric field of 2-

5v/cm until the tracking dyes reached the bottom of the

gel. DMA fragments were visualized by illumination with

short-wave UV light and gels photographed on Polaroid film

using a Polaroid MP-4 camera.

2.3.4. Autoradiography

Samples were autoradiographed at -70°C using

intensifying screens. Fuji film was sensitized by pre- flashing to an O D ^ j-q °f 0.1-0.3.

2.3.5. Gel filtration

Gel filtration was used to remove unincorporated radiolabelled nucleotides from nick translation reactions.

A pasteur pipette was plugged with polymer wool. A matrix of Sephadex G-50 was poured into this pipette and rinsed with 3 column volumes of 2 x SSC (SSC is 0.15M NaCl, 0.15M

43 Ma citrate). The sample was applied to the column in lOOul volume and the column washed with more 2 x SSC. Fractions were collected in 1.5ml eppendorf tubes, monitored and the excluded peak of radiolabelled DMA pooled.

2.3.6. Determination of radioactivity 32 P radioactivity was measured by Cerenkov counting at an efficiency of approximately 30%. 3 H radioactivity was measured by counting in toluene- based scintillant (0.5% w/v 2,5 diphenyloxazole [PPO],

0.03% w/v l,4-di-2-[5 phenyloxazolyl] benzene in toluene) diluted 4:1 in Triton X-100.

2.4. Specific methods

2.4.1. Preparing high molecular weight DNA from tissue

culture cells

Tissue culture cells were centrifuged for 5mins at

3000rpm in a Sorvall SA600 rotor and carefully resuspended in phosphate-buffered saline (PBSA - 0.15M NaCl, 3mM KC1,

4mM Na HPO^, , 1.5mM KH PO^) . Cells were washed a further time in PBSA and finally resuspended in 0.15M NaCl, 0.5% w/v sodium dodecyl sulphate (SDS) and lOOug/ml Proteinase

K. Cells were incubated overnight at 37°C then allowed to cool and extracted twice with phenol saturated with TE buffer and once with chloroform/isoamyl alcohol (24:1)

44 before ethanol precipitation and re-dissolution overnight o at 4 C. DMA concentrations were estimated by 2 GO measurements using a Cecil CE595 dual beam

spectrophotometer.

2.4.2. Small scale preparation of bacteriophage DNA

All host bacteria were harvested from an exponential

culture in L-broth (5g/l yeast extract, 10g/l tryptone,

5g/l NaCl, pH7.2) supplemented with lOmM MgSO^ and 0.4% w/v maltose and stored in lOmM MgSO^ at 4°C. Suitable

dilutions of the 'X-library' bacteriophage mix were pre­ adsorbed onto approximately 10^ bacteria and plated on tryptone plates (12g/l tryptone, 5g/l NaCl, llg/l agarose)

in a lawn of tryptone top agarose (12g/l tryptone, 5g/l

NaCl, 7g/l agarose) and incubated overnight at 37°C.

Single plaques were picked with a pasteur pipette into 1ml of TM buffer (lOmM Tris-HCl, pH7.5, 2mM MgCl^) and o 8 incubated for 4hrs-overnight at 4 C. Approximately 10 cells were preadsorbed v/ith 0.05-0.lml of this solution and plated in a lawn of tryptone top agarose on tryptone agarose plates. After overnight incubation at 37°C the plates which were almost confluently lysed were overlaid with 5ml of TM and the phage allowed to elute for l-2hrs at 4°C. The phage solution was transferred to 13ml centrifuge tubes and bacterial debris removed by centrifugation at 5500rpm in the Sorvall SAGOO rotor for lOmins at 4°C. The supernatant was digested with RNAase A and DNAase I at concentrations of lug/ml for 30mins at

45 o 37 C. Bacteriophage particles were precipitated by addition of an equal volume of a solution containing 20% w/v polyethylene glycol (PEG), 2M WaCl and incubation at

4°C for 2hrs-overnight. The precipitate was recovered by centrifugation at 6000rpm for 20mins at 4°C and removing the supernatant by aspiration and air-drying. Phage were resuspended in 0.5ml of TM by vortexing and debris removed by centrifugation at 5500rpm for 2mins at 4°C. The supernatant was transferred to a 1.5ml eppendorf tube to which 5ul of 10% w/v SDG and 5ul of 0.5M EDTA (pH8.0) were added. The solution was incubated at 68°C for 15mins to rupture the phage particles, allowed to cool and extracted once with phenol saturated with TE and once with chloroform/isoamyl alcohol (24:1). DMA was ethanol precipitated from the supernatant and resuspended in 50ul

TE. lOul of this solution was removed to a fresh eppendorf tube with 1.2ul restriction enzyme buffer and 1-2 units of enzyme. Fragments were analysed by agarose gel electrophoresis.

2.4.3. Large scale preparation of bacteriophage DMA

The method used to obtain larger amounts of bacteriophage DMA is essentially that described by

Blattner et al., (1977). A phage solution of a single plaque picked into 1ml of TM buffer was preadsorbed onto 8 approximately 2.5 x 10" cells m a lOmM MgSO^ suspension at 37°C for 15mins. These were then inoculated into 200ml

46 of L-broth supplemented with lOmM MgSO^ and 0.4% w/v

maltose and shaken vigorously overnight at 37°C.

Concomitant growth of bacteria and bacteriophage results

in lysis of the culture which was completed by addition of

5ml of chloroform. Bacterial debris was removed by centrifugation in 250ml GSA bottles in the Sorvall GSA

rotor at 8000rpm for 15mins at 4°C. 2ml MgC^# 7g NaCl and

25g PEG were added to the supernatant, dissolved and left overnight at 4°C for the phage to precipitate. PEG precipitates were recovered by centrifugation in the GSA

rotor at 7500rpm for 20mins at 4°C. The precipitate was air dried and taken up in 2ml of TM buffer. This high titre phage suspension was carefully layered onto a caesium chloride step gradient of 2.5ml of 1.7g/ml, 3ml of

1.5g/ml and 4ml of 1.3g/ml CsCl in a 13ml cellulose nitrate centrifuge tube. Phage were banded by ultracentrifugation in the Sorvall SW-40 swing out rotor at 32,000rpm for 2hrs at 20°C. Phage bands were extracted using a syringe and dialysed overnight at 4°C against TM buffer. The phage DNA v/as extracted by proteinase K digestion for 3hrs at 55°C followed by phenol and chloroform extraction, ethanol precipitation and re­ dissolution in TE buffer.

2.4.4. Labelling DMA by nick translation

The procedure for radio-labelling recombinant DNA probes v/as essentially that described by Rigby et al.

(1977). Trittiated deoxyribonucleotides came in 50%

47 ethanol and were dried down by vacuum and re-constituted 3 in 5ul of 11^0 by vortexing. II nick translation included 3 3 3 0.5ug of DNA, 50uCi each of H-dATP, H-dCTP and H-dTTP,

5mM MgC^/ 50mM Tris-HCl pH7.5, lOmM yS -mercaptoethanol,

4uM dGTP/ 5 units of endonuclease free E.coli DNA

polymerase I and 5-50pg of DNAase I in a 50ul volume. The

amount of DNAase I to be added was determined by sizing

reaction products on an alkaline agarose (denaturing) gel

and using the amount that gave a mean fragment size of

approximately lkbp. Incubation was for 150mins at 15°C

after which the reaction was quenched by gel filtration. 32 P nick translations were essentially similar except 3 2 25uCi of P-dCTP (>400Ci/mmol) was used as label and 4uM

dATP, dGTP and dTTP were used in the reaction.

Alternatively reactions used the Amersham nick translation kit. lOul of solution I and Sul of solution II were added 32 to 0.5ug DNA and 25uCi P-dCTP and water to a final volume of 50ul. Incubation times and gel filtration were

i d e n t i c a l .

2.4.5. Screening lambda recombinants

In situ hybridization to phage plaques v/as performed after the method of Benton and Davies (1977). The ' X- 2 library' was plated on 400cm bio-assay dishes in tryptone top agarose and plates v/ere chilled to 4°C. Sterile nitrocellulose filters were labelled with pencil marks, soaked in 3 x SSC for 30mins and blotted dry. Filters were

48 laid onto the surface of the plates, the first for 30secs,

the second for lmin, the third for 2mins and the fourth

for 4mins. Filters were then laid phage side uppermost for

3mins on a pad of Whatman 3MM paper soaked in 0.5M NaOH,

1.5M NaCl to denature phage DNA. Filters were then

submerged twice (30 secs each) in 0.5M Tris-HCl pH7.5,

1.5M NaCl and blotted dry. Filters were wrapped between

sheets of Whatman 3MM paper and baked for 2hrs at 30°C.

Filters were soaked for 30mins in 3 x SSC at 65°C and

overnight with shaking in 3 x SSC, 10 x Denhardt's

solution at 65°C. 1 x Denhardt's solution is 0.02% w/v

Ficoll, 0.02% w/v polyvinyl pyrrolidone and 0.02% w/v BSA

(Denhardt, 1966). The final pre-hybridization v/ash was for

2hrs in 3 x SSC, 0.1% w/v SDS, 0.1% w/v sodium

pyrophosphate, 10% w/v dextran sulphate, lOOug/ml

sonicated denatured herring sperm DNA, 5 x Denhardt's

solution at 65°C with shaking. The same solution was used

for hybridizations. Filters were transferred to hybridization chambers designed by A. Jeffreys, Leicester

University, to which was added 5-10ml of hybridization 32 . buffer and P-labelled probe DNA denatured by immersing

in boiling water for 5mins. Hybridization chambers were

left shaking overnight in a 65°C v/ater bath after which

filters were removed and washed 5 times in 3 x SSC, 0.1%

SDS, 0.1% sodium pyrophosphate for 5mins each at 65°C with

shaking. Filters were then transferred to 1 x SSC, 0.1%

SDS, 0.1% sodium pyrophosphate for two washes at 65°C of

30mins each. Filters were then blotted dry, covered in

49 clingfilm and transferred to X-ray cassettes for

autoradiography.

Phage which reacted positively with probe DMA were

picked with a pasteur pipette and propagated as plate

lysates. 2ul of each lysate were spotted on an ordered

array of 5 x 4 spots on a bacterial lawn and grown

overnight. These plates were then rescreened with the same

probe DMAs.

2.4.6. Southern transfers

Genomic or recombinant DMA for Southern transfers was

fractionated on 0.8% agarose gels. A hybridization marker

of lOOpg of Ilindlll digested lambda DMA was

electrophoresed in parallel. In addition visible size

markers of lug of Hindlll digested lambda DMA was run on

the same gel. This gives fragment markers of 23.1, 9.4,

6.6, 4.4, 2.3, 2.0 and 0.6 kbp. The lambda 'arms' of

recombinant phage hybridize to the largest four fragments.

After electrophoresis gels were photographed and denatured

for 2hrs in 0.5M NaOM, 1.5M MaCl. Gels were then

neutralized in 0.5M Tris-HCl pH5.5, 3M MaCl for 2-4hrs

with several changes. DMA was blotted overnight in 20 x

SSC onto a nitrocellulose filter by capillary action using

the apparatus described by Southern (1975). The

nitrocellulose filter was washed for 20mins in 2 x SSC,

blotted dry and baked for 2hrs at 80°C. Filters were pre­ hybridized and hybridized as described in section 2.4.5.

50 with an additional post-hybridization wash in 0.1 x SSc,

0.1% SDS, 0.1% sodium pyrophosphate for 30mins at 65°c

with shaking as necessary.

2.4.7. Competing repetitive sequences in Southern blot

hybridizations

The method used to reveal the cognate band in a total

genomic Southern blot probed with a cloned fragment

including a repeated sequence is based on that of Davies

et al.(1933B). Total human DMA was sheared to an average

length of 2kbp by sonication. The sheared DMA was mixed 32 with p-labelled probe DMA to a final concentration of

O.lmg/ml in 3 x SSC. Driver and probe DMA v/ere denatured together by boiling for 5mins and then transferred

immediately to 65°C for an incubation of 2-4hrs. The DMA was then transferred to hybridization chambers to which hybridization buffer constituents had been added to give a final volume of 5-10ml. Hybridization and post­ hybridization washes were as described in 2.4.5.

2.4.8. Preparation of metaphase spreads

The method for preparing slides of human metaphase chromosomes was essentially that of Moorhead et al.

(1960). 10ml of blood were collected in a heparinized tube and the red cell layer allowed to settle. 0.3-0.4ml of the white cell layer was added to 10ml of RPMI-1640 medium supplemented with 10% v/v foetal calf serum and 0.1ml of a solution of phytohaemagglutinin. 2-3 drops of the red cell

51 layer were added before careful mixing and incubation overnight at 4°C. Cultures were then transferred to 37°C and incubated for 72hrs, mixing daily by inversion. 0.15ml of a 25ug/ml solution of colchicine was added 90mins before removal from the 37°C incubator. Cells were harvested by centrifugation in a Sorvall bench centrifuge at 1500rpm for 5mins. Cells were carefully resuspended with constant agitation in 0.075M KC1 prewarrned to 37°C and then incubated for 5mins at 37°C. Osmotically swollen cells were recovered by centrifugation at 1500rpm for

5mins and fixed v/ith 10ml of freshly prepared pre-chilled

(on ice) fixative (1 part glacial acetic acid to 3 parts methanol) by careful resuspension with constant agitation.

After leaving at 4°C for at least 30mins, cells were pelleted by centrifugation at 1500rpm for 5mins and washed twice in freshly prepared fixative before resuspending finally in 0.5-2ml. This suspension was spread on slides which had been cleaned by soaking overnight in chromic acid and rinsing under the tap for at least 30mins. 1 or 2 drops were applied to the centre of the wet slides and allowed to spread before drying on a hot plate. Spreading was monitored by phase contrast microscopy of dried s l i d e s .

2.4.9. G-Banding of metaphase chromosomes

Two methods of banding were employed, the first using trypsin/Giemsa (Seabright, 1971) and the second

52 Lipsol/Leishmann' s (Malcolm et al., 1982). Trypsinization

of slides was achieved by soaking slides for 30secs-

2.5mins in a 1:14 dilution of Trypsin in 0.9% w/v NaCl at

4°C. Slides were then rinsed thoroughly in 0.9% w/v NaCl,

air dried and stained in coplin jars with 5% w/v Giemsa in

phosphate buffer pH6.8 for 40mins. Well banded metaphase

spreads were recorded by noting microscope vernier

positions and photographed. The alternative and preferred

banding procedure was to immerse slides for 5-60secs in a

1:100 v/v dilution of Lipsol detergent in water, rinse off

with 0.9% NaCl and stain by covering with 5ml of

Leishmann's stain diluted 1:5 in phosphate buffer, pH6.8.

Leishmann's stain was made by dissolving 1.5g of powder in

a litre of acetone-free methanol, stirring for 2hrs and

filtering through 2 layers of V7hatman 3MM filter paper.

Slides were stained for 2.5mins and then rinsed with

phosphate buffer, pII6.8 and dried on a hot plate. Well

banded spreads were recorded and photographed.

2.4.10. 'In situ' hybridization

The area of spreads was marked with a diamond pencil

and photographed slides were destained by successive dehydration in 50%, 75%, 95% and 100% ethanol for 2mins

each and allowed to dry. Slides were then floated in trays in a 37°C waterbath and allowed to warm up before placing

0.2ml of a solution of RNAase A (O.lmg/ml in 2 x SSC) onto each slide and covering with 24 x 50mm coverslips previously cleaned in 0.1M IIC1 and distilled water. After

53 an incubation of lhr the coverslips were removed and the

slides rinsed three times in 2 x SSC for 2mins each before

dehydration in 50%, 75%, 95% and 100% ethanol for 2mins

each. Slides were then air dried and returned to trays in

a 70°C waterbath and allowed to warm up. 0.2ml of

denaturing solution (70% v/v forrnamide/2 x SSC) was then

placed onto each slide under acid cleaned 24 x 50mm

coverslips. After 2mins of incubation slides were again

rinsed three times in 2 x SSC, dehydrated and air dried.

Trittiated, nick translated probe was prepared as

described in 2.4.4. Pooled excluded fractions were ethanol

precipitated, re-dissolved in 20ul of formamide hybridization buffer and counted. Formamide hybridization buffer is 50% v/v formamide/2 x SSC/10% w/v dextran

sulphate/5 x Denhardt's solution/0.lmg/ml sheared, denatured herring sperm DNA/0.1% w / v sodium pyrophosphate.

By correlating the recovered radioactivity with the

initial excluded fractions, the labelled DMA was diluted to a concentration of 0.1-10ug/ml in formamide hybridization buffer. lOul of this dilution was then spotted onto slides and covered with acid cleaned 22 x

22mm coverslips ensuring that no air bubbles became trapped underneath. The edges of the coverslip were sealed with Holdtite adhesive and slides were incubated on trays in a 42°C waterbath overnight. The adhesive was removed with tweezers and coverslips were soaked off in 2 x

SSC/0.1% sodium pyrophosphate. Slides were washed 5 times

54 in 2 x SSC/0.1% sodium pyrophosphate for 2mins each and 3

times in 0.1 x SSC/0.1% sodium pyrophosphate for 2mins

each at room temperature. Slides were then transferred to

0.1 x SSC/0.1% sodium pyrophosphate at 55°C for two 30mins

washes and finally washed extensively in 0.1 x SSC/0.1%

sodium pyrophosphate overnight with stirring at 4°C.

Slides were dehydrated, air dried and coated v/ith a

subbing solution of 0.1% w/v gelatin, 0.01% w/v chromalum

by dipping for 30secs. After allowing to dry, slides were

coated with a layer of emulsion (Ilford K2 or L4 or Kodak

MTB-2) diluted 1+1 with water at 50°C by dipping for

approximately 20secs. Emulsion was wiped from the back of

the slides before they were dried vertically in test tube

racks in the dark room for l-2hrs. Autoradiography was

performed in light-tight boxes with dessicant at 4°C for

10-22 days. Slides were developed in Ilford Phenisol diluted 1+4 with water for 5mins, rinsed in water and

fixed in Ilford Hypam diluted 1+4 with water for 3mins.

After rinsing under the tap for 30mins, slides were air dried and stained with Leishmann's stain diluted 1+4 v/ith phosphate buffer, pH6.8, for 3mins.

2.4.11. Scoring hybridized spreads

Recorded spreads were compared after autoradiography to the photograph of the G-banded spread before hybridization. The position of grains on the autoradiograph v/as marked directly onto the G-banded karyotype of each spread and totals for each chromosome

55 computed by summation over all recorded spreads. This

observed value was compared to an expected value computed

by multiplying the total number of chromosomes over all

scored chromosomes by the diploid percentage DMA content

of each individual chromosome. For a random distribution

of grains the deviation of each observed chromosomal grain

value from the expected (0-E) will be normally distributed

about a mean of zero. Consequently the significance of

each result can be estimated by comparing the number of

standard deviations each 0-E value takes to the

standardized normal distribution.

Grain distribution was also displayed

diagrammatically by using chromosome ideograms to display

composite distribution. The position of each grain as

marked on the G-banded karyotype was marked on a single

karyotype ideogram. In this way the total number of grains

over individual G-bands or interbands and hence the intra-

chromosomal distribution was also determined.

2.4.12. Pedigree analysis

Linkage between markers was estimated by the

sequential or lods method (Morton, 1956). Recombination

fractions for any pair of loci were estimated by

interpolation after summing the lod scores over all the families determined from a standard lod score table (Race and Sanger, 1975). Additionally, lod scores were computed by using the program LIPED (Ott, 1974).

56 CHAPTER 3

Results

57 3.1. Screening recombinant bacteriophage 'in situ'

The majority of non-repetitive human DMA is organized

in discrete blocks interspersed with repetitive elements.

These repeated DMA sequences are only weakly conserved between species and consequently we predicted that

recombinant phage from the genomic library enriched for

the X would react with a total human repetitive DMA probe

and only weakly with a mouse probe. Therefore

reassociation of a repetitive DMA probe from a human x mouse somatic cell hybrid whose sole human chromosome was

the X with recombinant phage could be attributed to repetitive sequences present on the human X chromosome.

Similarly, experiments utilizing radiolabelled somatic cell hybrid DMAs containing different amounts of human X chromosomal DMA should delineate recombinant phage specific to a particular portion of the X chromosome.

A few hundred plaques from the X enriched library were transferred to nitrocellulose by the Benton-Davis 32 procedure and incubated with P-labelled total genomic

DMAs. Under these conditions, only the repetitive DMA in the probe is in sufficient concentration to allow autoradiographic detection of reassociated DMA. To ensure the reaction was species specific, an initial comparison was made between human and mouse DMAs as probe. IIorl9X is a somatic cell hybrid containing the X as its sole human chromosome (Goodfellow et al., 19H0) and is derived from the mouse cell line IR Radiolabelled DMA from male human blood and from these cells was hybridized to duplicate

Benton-Davis filters (see Materials and Methods). Fig. 1.

shows the autoradiographs after a second screening of

recombinants picked from the first screening for giving

1., a weak signal with Horl9X DMA and with mouse, 2., a

weak signal with Horl9X DMA and none with mouse, 3., a

strong signal with Horl9X DMA and a weak signal with mouse

and 4., a strong signal with Horl9X DMA and none with

mouse. The spots on the autoradiographs corresponding to

recombinants DXR1.5 and DXR4.4 are blank as these

recombinants gave a zero titre on this plate. There were

no recombinants that gave a signal with mouse but not

human DMA as the probe. The probes in each secondary

screen were in A; human DMA, B; mouse DMA and C; Horl9X

DMA. The result shows that none of these recombinants

reacts as strongly with horl9X DMA as with human DMA. This

can be attributed to two factors, firstly that the X

chromosome concentration in each probe is different; the hu ma n iprobe has one X chromosome in 46 hu ma n chromosomes whilst the IIorl9X probe has one X chromosome in a background of approximately 60 mouse chromosomes (Davies

et al . , 1981). Secondly, the concentration of individual

repeat families will be higher in the human probe due to

the presence of autosomal copies of each repeat. The

result additionally shows that IIorl9X DMA always hybridizes to recombinants the human DMA reacts with whilst the mouse probe does not. This confirms the species

59 c

Fig. 1 Recombinants from the 'X - enriched* library- screened with total genomic DNA from : A - Human blood; B

- mouse fibroblasts; C - Horl9X fibroblasts

60 specificity of many repetitive sequences and implies a good representation of X chromosomal DMA sequences in the

library. That conserved repeats also exist is demonstrated by the recombinants which cross react with mouse DMA. The pattern is confirmed by taking the individual recombinants and hybridizing to Southern (1975) filters of restriction endonuclease digested hybrid DMAs. Autoradiographs show an

intense smear over the human, Horl9X and mouse DMAs for

the recombinants DXR1.3. and DXR1.4. but only on the human

and Morl9X tracks with the recombinants DXR2.3. and

DXR2.5. (Results not shown). The human x mouse somatic cell hybrid MCP-6 retains

the translocation chromosome t(S;X )(6qter-p21::Xql3-qter)

as its only human chromosome (C-oodfellow et al., 1902). Me

reasoned that a comparative screening between total DMA

from this hybrid and that from Horl9X should delineate

repeats on the X chromosome from Xpter-ql3 on the basis of

their reaction with IIorl9X and not MCP-6 DMA. Duplicate . . . 39 filters were hybridized overnight to ^P-labelled total hy br id DM a at concentrations adjusted to compensate for

the different ploidies of the hybrids. Filters were washed

to a stringency of 1 x SSC at 65°C and exposed to Fuji X-

ray film with an intensifying film at -70°C for two weeks.

Fig. 2. shows the autoradiographs which demonstrate no

visible differences. Attempts to superimpose negatives

failed to demonstrate any differences. This result implies

there are no repetitive sequences in the X-enriched

library which are unique to the region Xql3-pter.

61 Fig.2 Two duplicates of one quarter of a 20 x 20cm nitrocellulose filter blotted to a bio-assay dish of several thousand recombinants from the 'X-enriched library'. The probe in each hybridization is total DNA from A - Horl9X;B - MCP-6 cells.

62 3.2 Isolation and characterization of

individual recombinant bacteriophage

To facilitate the identification of X chromosome DMA

sequences it is important to isolate them from any

adjacent reiterated sequences which may have become cloned

within the same recombinant molecule. Consequently we

reasoned that recombinants on Benton-Davis filters which

did not hybridize to total human DMA would contain only

non-repetitive sequence and therefore be most suited to

further characterization. Unfortunately phage picked on

this basis largely turned out to carry DMA derived from

bacteriophage lambda inserted between the left and right

arms of the cloning vector, or no insert at all and an

abnormally large vector arm. As a consequence, the

stochastic approach of picking plaques at random from the

library was adopted. The rationale for this procedure is

that single copy sequence would almost certainly be

contained in the same molecule as repetitive sequence and

so a more representative collection of X chromosome DM.A

sequence is accomplished. Such interspersed non-repetitive

DMA can be detected by restriction enzyme digestion and

re-cloning or by competition in hybridization with a

repetitive probe.

Individual plaques were picked, propagated on a

suitable host strain and the DMA extracted from plate

lysates (see Materials and Methods). The DMA was digested i 63 Fig .3 Polaroid photograph of an a g arose gel with

EcoRI digested DNA f rom s everal d i ffer ent recomb inants

derived from the ' X- enriched ' library e lectrophoresed in

l E I-b u ffe r overnight . Lanes 1 and 14 are lambda DNA

d i gested with HindII I which g ives mo lecular weight marker

bands of 23.1, 9.4 , 6. 6 , 4 . 4 , 2 . 3 , 2. 0 and 0 . 6kbp . The

recombi nant s whose DNA has been f ractio nated a r e from

Lanes 2 - 13; DX l - 12.

• 64 by the restriction endonuclease EcoRI, fragments were

separated by agarose gel electrophoresis and visualised by

ethidium bromide fluorescence. Fig. 3. is a photograph of

a gel of EcoRI digests of the clones DX1-12. The largest molecular weight bands represent the DMA of the vector

phage ('arms') of 21.7kbp and 13.3kbp. The smaller bands

are the human DMA inserts ranging from 1.7kbp (DX9) to

8.4kbp (DX7). The clones DX1 and DX3 have no detectable

DMA insert and a concomitant extended long 'arm'. The

presence of more than one insert in DX9 suggests either that the EcoRI digestion of sorted chromosome was

incomplete or that fragments had religated together prior

to ligation to bacteriophage lambda DMA..

The reiteration frequency of human DMAinsert

sequence was examined by Southern transfer of gels such as

Fig. 3. to nitrocellulose and overnight hybridization to

3 p total human DMA labelled with “P by nick translation (see

Materials and Methods). Fig. 4. shows the autoradiographs of such an experiment. In A the filters have been washed

to a stringency of 3 x SSC at 65°C, in P> to 1 x SSC at

65°C and in C to 0.1 x SSC at 65°C(see Materials and

Methods). The lambda DMA bands are retained on the

autoradiographs to act as markers by inclusion of a trace 32 . amount of P-labelled lambda DMA m the hybridization

reaction. It is known that reassociated repetitive DMA has

a wide melting range (Britten and Kolme, 1968) and by

reducing the salt concentration and hence the Tm of

65 X DX12 3 4 5 6 7 8 9 10 11 12 X

XDX1 2 3 4 5 6 7 8 9 10 11 12 X

Fig.4 - A Southern blot hybridization of DMA from recombinants DX1 - 12 hybridized to both lambda DMA and total human DMA. The filter was washed after hybridization to a stringency of 3xSSC. B - The same filter after washing to a stringency of IxSSC.

6 5 X DX123 4 5 6 7 8 9 10 11 12 X

X DX1 2 3 U 5 6 7 8 9 10 11 12 X

Fig.4 - C The same filter in Fig.4A washed to a stringency of O.lxSSC. D - A duplicate filter hybridized only to lambda DMA.

67

( reannealed Did A (Britten et al. / 1974) it is possible to selectively melt off imperfect hybrids. Thus in Fig. 4 . A

and 8, DX4 and DX7 still reanneal to a significant component of human DMA but at a stringency of 0.1 x SSC at

65°C/ hybrids are not formed, as shown in Fig. 4C. These

recombinants are candidates for non-repetitive DMA

sequences, whilst those such as DX2 or DX5 clearly contain

repetitive DMA sequences. The result of hybridizing lambda

DMA alone to a duplicate filter is displayed in Fig. 4D

and clearly only the high molecular weight bands of the vector arms are detected. The smear of radioactivity at

the bottom of the autoradiograph represents either vector

DMA degraded by nucleases or bacterial DMA reacting with trace amounts in the probe.

In total, 23 clones were examined in this fashion. A summary of the data from these clones is presented in

Table 1. Six of the 23 do not contain any human DMA and

the average insert size of the remaining 22 is

approximately 6.5kbp. In total the cloned DMA examined

amounts to 142kbp.

3.3. Chromosomal localization I. Somatic cell hybrids

Having isolated human DMA sequence from the X

enriched genomic library it is necessary to show that it

is derived from the human X chromosome. The most practical

method to employ is the use of somatic cell hybrids which

68 Table 1

Recombinant DNA Insert (kbp) Hybridization to total human DNA

DX1 no insert, extended long arm DX2 7.2 + DX3 no insert, extended long arm DX4 4.1 DX5 3 .8 + DX6 7.7 + DX7 8.4 — DX8 6.1 + DX9 5.2, 1.7 + DX10 7.9 + D X 1 1 5.9 + D X 1 2 5.9 + DX13 2.2, 1.8 - DX14 8.0 + D X 1 5 6.5 - D X 1 6 lambda B insert D X 1 7 5.5 + DX18 6.2 + D X 1 9 lambda insert DX20 7.3 + D X 2 1 lambda inserts DX22 6.6 + DX2 3 6.7 + DX24 7.2 — DX2 5 5.6 + DX3 3 7.4 + DX48 7.2 + RE 3 lambda B insert

Summary of results obtained from the analysis of

randomly isolated recombinants from the X-enriched

library. Those recombinants marked + retained an

autoradiographic signal after hybridization to total human

DNA and washing the filter to 0.1 x SSC.

i 69 contain the X chromosome as their sole human complement.

Such hybrids have been constructed by Goodfellow by

selecting for the X- li nk ed 1IGRPT enzyme in HAT medium. The

fibroblast cell line Horl9X retains an intact X chromosome

in a tetraploid mouse background (Goodfellow et al./

1930), MCP-6 is a teratocarcinoma cell line and carries

the translocation chromosome t(6;X )(6qter-6p21::Xql3-qter)

in a normal diploid mouse background (Goodfellow et al.,

1932), IUI-5 is a fibroblast line with a single X

chromosome deleted for Xpll-pter in a diploid mouse

background (Solomon et al., 1976) and IR is the mouse

fibroblast line from which Horl9X and IbI-5 were

constructed. Cells were grown in tissue culture and the

DMA extracted for digestion with the restriction

endonuclease EcoRI (see Materials and Methods). Digests

were fractionated by agarose gel electrophoresis and

visualized by ethidium bromide fluorescence. A photograph

of such a gel is shown in Fig. 5. with lambda DMA digested

with Hindlll in lane 1 acting as molecular weight markers.

The intensely fluorescing bands present in the smear of

DMA fragments represent a family of repetitive DMA

sequences organized in tandem arrays, each copy containing

a single EcoRI site (Darling et al., 1932; Mu and

Manuelidis, 1930). Restriction fragments were transferred

to nitrocellulose by the method of Southern (1975) and

filters were hybridized overnight to recombinant DMA 3 2 labelled with P by nick translation (Rigby et al.,

i 70 Fig.5 Polaroid photograph of an agarose gel of EcoRI digested DNAs from : Lanes 2 and 7, human blood; Lanes 3 and 8, Horl9X fibroblasts; Lanes 4 and 9, IWI-5 fibroblasts; Lanes 5 and 10, MCP-6 teratocarcinoma cells;

Lanes 6 and 11, mouse fibroblasts. Lane 1 contains lambda

DNA digested with HindiII electrophoresed in parallel.

71 1976). Filters were washed to 1 x SSC or 0.1 x SSC at 65°C depending on the background hybridization of imperfect hybrids. Clearly, recombinants which hybridized to a single EcoRI band in the human and Horl9X but not the mouse (IR) tracks must be derived from the human X chromosome. Furthermore, those recombinants which hybridized additionally to single bands in the IVTI-5 and

MCP-6 tracks must be derived from Xql3-qter, those which hybridized to IVII-5 but not MCP-6 from Xpll-ql3 and those which hybridized to neither (but did hybridize to HorlPX)

from Xpll-pter.

Some of the chromosomal localizations effected by this method are shown in Figs. 6-8. where a series of autoradiographs are shown. Fig. 6.A shows that the recombinant DX24 hybridizes to a 7.0kbp band (and additionally but less intensely to a 7.8kbp band) in the human track alone. This implies that it is derived from a human autosome rather than the X chromosome. This autosome is most likely to be either or chromosome 8 as these chromosomes are closest in size and DHA content to the X and are fractionated by the flow sorter into the same group as the X (Davies et al., 1981). It is unlikely to be chromosome 6 as the translocation chromosome in MCP-

6 carries most of chromosome 6. In Fig. 6B the autoradiograph demonstrates that the recombinant RD6 hybridizes to a 5.3kbp band in the human track which is also present in the Horl9X and IbI-5 tracks but absent

from the MCP-6 and IR tracks. This indicates that this

72 A B

1 23456 1 2345 6

9 ♦ * * * ‘ + % *

Fig.6 Southern blot hybridizations of EcoRI digests of DNA from : Lane 2; human blood, Lane 3; Horl9X fibroblasts, Lane 4; IWI-5 fibroblasts, Lane 5; MCP-6 teratocarcinoma cells, Lane 6; mouse fibroblasts to A.

DX24, B RD6.

73 recombinant is human in origin (as it does not hybridize

to mouse DLJA) and derived from the X chromosome in the

region Xpll-Xql3. The higher molecular weight bands evident in the human track represent imperfect duplexes between homologous sequences. These can be melted by increasing the stringency to 0.05 x SSC at 65°C.

Fig. 7.A shows the result of hybridizing the recombinant S21 to the somatic cell hybrid panel. A single

7 .3kbp band is detected in all tracks bar the mouse parent

(IR) and this indicates that the recombinant is derived from a locus on the human X chromosome between Xql3-qter.

The same is true for the recombinant DX4, as Fig. 7.B demonstrates. This hybridizes to a 4.2kbp human fragment

(and an additional 2.2kbp band revealed on longer exposure) present in all tracks bar the mouse.

The overexposed autoradiograph shown in Fig. o.A demonstrates that the recombinant DX7 hybridizes to all the tracks, including the mouse, but in a species specific fashion. This recombinant detects an 3.4kbp band in the human track that is absent from all the hybrid tracks and the mouse but, in addition, detects a 2.2kbp band (and also a 1 .3kbp band revealed on longer exposure) present in all the hybrids and the mouse but not human tracks. This fragment must then be derived from a human autosome and contains sequence which is conserved between mouse and human but contained within diverged EcoRI sites.

In Fig. 8.B, DX6 detects a single 7.7kbp band present

74 A B

Fig.7 Southern blot hybridizations of EcoRI digests of DNA from : Lane 2; human blood, Lane 3; Horl9X fibroblasts, Lane 4; IWI-5 fibroblasts, Lane 5; MCP-6 teratocarcinoma cells, Lane 6; mouse fibroblasts to A S21 and B D X 4 .

75 A B

Fj.g.8 Southern blot hybridizations of EcoRI digests of DNA from : Lane 2; human blood, Lane 3; Horl9X

fibroblasts, Lane 4; IWI-5 fibroblasts, Lane 5; MCP-6

teratocarcinoma cells. Lane 6; mouse fibroblasts to A DX7

and B D X 6 .

76 in all tracks except the mouse and is therefore derived

from Xql3-qter. This 7.7kbp fragment includes a highly reiterated sequence (of unknown origin) as evidenced by the reaction v/ith total human DMA in Fig. 4. and under normal conditions hybridizes to a smear of fragments on a

Southern blot of genomic DMA. This repetitive sequence can be competed out of hybridizing to the filter by hybridizing the nick translated recombinant v/ith total human DMA in solution before and during hybridization to the filter (see Materials and Methods). In this way, the cognate EcoRI fragment representing the single chromosomal locus from which the recombinant is derived can be detected as shown without physical separation from reiterated sequences present on the same molecule.

In most of the Southern blots, a background smear of radioactivity can be seen in the human and hybrid tracks

(although very much fainter in the hybrid). The cause of this smear is interpreted as hybridization of a reiterated sequence present on the same recombinant molecule to many homologous sequences in the genome. The evidence for this interpretation lies in the species specificity of the smear which is rarely evident in the mouse track.

A summary of the chromosomal localizations effected by this method is presented in Table 2. A higher resolution using a hybrid panel with many more derivative

X chromosomes has been possible in the laboratory of

Ropers. Experiments in that laboratory have further localized DX13 to Xq23-qter, RB6 to Xq26-q27, RC3 to Xpll-

77 Table 2

Recombinant Hybridization to Localization

HUMAN H0 RL9X IWI-5 MCP-6

DX4 + + + + X q l 3 - q t e r DX6* + + + + X q l 3 - q t e r DX7 + - - - autosomal D X 1 3 + 4* + X q l 3 - q t e r D X 1 5 + - -- autosomal DX24 + --- autosomal DX48 + + + + X q l 3 - q t e r RB6 + + + + X q l 3 - q t e r RD6 + + + - X p l 1 - q l 3 RC8 + + - - X p l 1 - p t e r LI .28 + - - X p l 1 - p t e r DP 31 + + + + X q l 3 - q t e r S21 + + + 4- X q l 3 - q t e r 52A + + + + Xq l3 - q t e r

Summary- of chromosomal localizations using somatic

cell hybrids.

* DX6 contains repetitive sequence and the

hybridization profile shown represents the cognate EcoRI

fragment and not other homologous sequences. i 78 Xp223, and 821 to Xq213-q26 (V:ieacker et al., 1983). Using

their hybrid panel in this laboratory, DX4 maps to Xq28- qter (results not shown).

In all these experiments hybridization to a single

EcoRI fragment of the same molecular weight as the cloned

fragment (compare, for example, Figs. 7 and 8 to Fig. 3.) was taken as evidence for a single genomic locus. This remains, of course, an assumption, but the assumption is

strengthened by the greater resolution of chromosomal

localization afforded by 'in situ' hybridization.

3.4. Chromosomal localization 11. 1 In situ1 hybridization

h Introduction

The technique of 'in situ' hybridization offers the greatest potential in ma mm al ia n gene mapping ye t it remains shrouded with s u s p i c i o n . It is clear that the reaction of input labelled DMA with chromosomal DMA on microscope slides yields specific DMA hybrids (Join e s ,

1973; Wimber and Steffensen, 1973). The problem is trying to detect a single locus corresponding to extremely small amounts of DMA. One can begin to approach the problem mathematically in the following fashion.

1. Let the input DMA be a trittiated 6kbp DMA sequence

labelled to a specific activity of 4.5 x 10 dpm/ug. This

is hybridized to metaphase chromosomes and exposed to

79 emulsion for 15 days

2. 1 mole of base pairs has a mass of approximately 660g . . . 7 Consequently a specific activity of 4.5 x 10 dpm/ug 7 G —"5 corresponds to 4.5 x 10 x 660 x 10 = 4.9 x 10 dpm/bp

6.023 x 1 0 23

3. By hybridizing 5S RNA to Drosophila salivary chromosomes Szabo et al. (1977) have estimated that only

9% of sites become label led. As the ratio of input to target DMA can reach as 1 ow as 100, it is unlikely that saturation is achieved, but assuming saturation, the amount of radioactivity at each site will be _ p _ ^ 9 x 5 x 103 x 4.9x 10 = 2.2x10' d pm/site 100

As there are two sites on every chromosome, this will be

4.4 x 10 dprn/chromosome

4. lode rn nuclear emulsions have an autoradiographic efficien cy for trittium of approximately 0.12 grains per beta d i s integration (Ada et al., 1966), thus for a 15 day exposure one would expect

15 x 24 x 60 x 0.12 x 4.4 x 10 =0.12 grains/chromosoine

This fig ure must be taken with extreme caution but clearly represen ts an experiment which becomes feasible with some enhancem ent. Such enhancement can be obtained by using double stranded (nick translated) DMA probes in conjunct ion with dextran sulphate in the hybridization reac tion (Wahl et al., 1979; Harper and Saunders, 1931).

Dextran sulphate accelerates the renaturation of a variety of DMAs approximately 10 fold in solution (I’etmur, 1975).

80 This is attributed to the polymer excluding the DMA from its volume and hence causing an increase in the effective

DMA concentration. For a bimolecular reaction, the ten

fold increase in rate corresponds to an increase of approximately three fold in the effective DMA concentration of each component. Thus when one component is immobilized a 3-4 fold increase has been observed with a single stranded probe (Wahl et al.f 1979). However, when the probe is randomly nicked, double stranded DMA (dsDUA), there is up to a hundred fold increase in rate and this dramatic increase is attributed to the ability of the dsDMA probe to form extensive networks (Dahl et al.,

1979). These observations have led to the development of an 'in situ' hybridization protocol that has general applicability to any

Saunders, 1931) which

(Szabo and Wahl, 1932).

The protocol used methods (Harper and Saunders, 1931; Malcolm et al., 1932) and direct experience (see Materials and Methods). The fidelity of the Paddington protocol was proven by the localization of a random genomic sequence derived from a library of DMA enriched for chromosomes 3, 4, and 5 by flow sorting. This recombinant, 4C11, contains two contiguous EcoRI fragments for a total of 6kbp and was nick translated to a specific activity of 4.5 x 10^ dpm/ug using three trittiated deoxyribonucleotides. The labelled

DMA was hybridized overnight to male metaphase spreads at

81 B

Fig.9 Human male metaphase spread A - after G-banding and B - after hybridization to 4C11 and autoradiography.

One of the two chromosome 6s is labelled.

82 a concentration of lOug/ml and exposed to Ilford K2

emulsion at 4°C for 15 days before developing and scoring

(see Materials and Methods). Fig. 9. is an example

autoradiogram, shown next to the same spread after banding

but before hybridization. The high background of grains is

evident, but clearly one chromosome 6 is labelled.

Table 3 compares the observed number of grains to an

expected value assuming random grain distribution. This

value is computed by multiplying the total number of

grains by each individual chromosome's percentage DMA

content of the diploid total (Mendelsohn et al., 1973). In

this instance only chromosome 6 is labelled significantly

over the 9 cells examined. Fig. 10 depicts the

distribution of grains over the 10 chromosomes scored

diagramatica1ly and clearly the recombinant is derived

from a sequence at 6pl2 as 18 of the total 39 grains lie

over this region. This result places into doubt the

fidelity of either the flow sorting from which 4C11 was derived, or the 'in situ' hybridization experiment. That

the latter is not the case is confirmed by hybridization of 4C11 to a somatic cell hybrid DMA panel identical to

those of Figs. 4-6. Fig. 11. shows the result of this

experiment which demonstrates that 4C11 detects EcoRI

fragments in only the human and MCP-6 DMA tracks. As MCP-6 contains the translocation chromosome t(6;X)(6qter- p21 : : Xql 3-qter) and 4C11 hybridizes to neither IIorl9X (X) nor IbI-5 (Xpll-qter) DMAs, this confirms the 'in situ'

0 3 Table 3

4C11 hybridised to 9 male metaphase spreads

Chro mo so me 0 E 0-E 1 15 23.11 - 3.11 2 18 22.52 - 4.52 3 20 18.62 1.38 4 9 17.82 - 8.82 5 9 17.08 - 8.08 6 43 16.12 26.88 7 21 14.78 6.22 8 21 13.61 7.39 9 11 12.65 - 1 .65 10 13 12.43 0.57 11 11 12.70 - 1 .70 12 7 12.54 -5.54 13 11 9.93 1.07 14 12 9.61 2.39 15 11 9.02 1 .98 16 6 8.27 - 2.27 17 6 7.95 - 1 .95 18 5 7.47 - 2.47 19 6 5.76 0.24 20 5 6.40 - 1 .40 21 3 4.38 -1.38 22 2 4.59 - 2.59 X 11 7.20 3.80 Y 1 2.45 - 1 .45

S D = 6 .88247

The deviation of observed (o) from expected (E) values (O-E) is estimated as a probability that the deviation is random by the number of standard deviations of all 0-E values each 0-E value takes. Only in the case of chromosome 6 is the 0-E value greater than one standard deviation. The probability that this value is random is approximately 0.005%.

84 X4CII 18 Chromosomes

6 p 1 2

Fig.10 Distribution of grains over 18 chromosome 6s

following 'in situ' hybridization to 4C11. The vertical

axis represents the composite number of grains and runs

from 0 to 20. The horizontal axis represents a G-banded

chromosome 6 running from the tip of the short arm at the

left to the tip of the long arm at the right. The banding

pattern is from the Paris Conference, 1981 (ISCN, 1981).

> 35 12 3 4 5

Fig.11 Southern blot hybridizations of EcoRI digests of DNA from : Lane 1; human blood, Lane 2; Horl9X fibroblasts, Lane 3; IWI-5 fibroblasts, Lane 4; MCP-6 teratocarcinoma cells, Lane 5; mouse fibroblasts to nick translated 4C11. The open arrows represent molecular weight sizes of 23.1, 9.4, 6.6 and 4.4kbp.

86 result. In this experiment if saturation of available

sites were accomplished, one would have expected 0.12

grains/metaphase chromosome. As the observed average is 1

grain/metaphase chromosome clearly dextran sulphate is

enhancing the rate and the dsDMA probe is facilitating

network formation.

The distribution of repetitive sequences in the human

X chromosome (and indeed throughout the genome) is clearly visualized in single cells by 'in situ' hybridization.

Fig. 12. demonstrates the distribution of two types of middle repetitive sequence. In A the probe is derived from

a 340bp or 630bp reiterated sequence homologous to the

African Green nonkey alpha satellite (Darling et al.,

1932), and in 3 the probe is derived from a 1.9kbp

dispersed repeat with a single Hindlll site (Manuel idis

and Biro, 1982; Crampton, unpublished observations). The

result indicates the remarkably different chromosomal

organizations of these two mainland middle repetitive

sequences. The HindiII 1.9kbp repeat is dispersed

throughout the genome with no obvious preferential sites.

Tnis is similar to the distribution of Alu sequences

revealed on hybridizing a cloned member, BLUR8 (Jelinek et

al., 1930), to metaphase spreads (results not shown). The

alpha RI repeat, however shows a striking constraint to

procentric locations, [particularly on chromosomes 1, 3, 5,

16 and 19, with some interstitial grains. The X chromosome

appears to be somewhat underrepresented for this

particular DMA sequence but a 2kbp BamHI repeat (Yang et Fig.12 Autoradiographs of human metaphase spreads hybridized 'in situ' to cloned human repetitive DNA sequences. In A the probe is alpha RI repetitive sequence and in B a copy of the 1.9kbp Hindlll repeat.

88 al., 1982; Willard et al., 1982) reveals X centromeric grains almost exclusively (results not shown).

B Cytological mapping of the X chromosome

Having proven the fidelity of 'in situ' hybridization with the localization of 4C11 to 6pl2, the X chromosome markers isolated and characterized as described in sections 3.2.-3.3. have been used to construct a high resolution cytological map of the X chromosome. Technical amendments were applied to the protocol with different experiments, principally in adjusting the probe concentration to reduce the rather high background evinced in Fig. 9. In addition, the trypsin-Giemsa banding used prior to hybridization for chromosome identification in the 4C11 experiment was abandoned in favour of Lipsol banding followed by Leishmann's stain (Malcolm. et al.,

1982). This modification resulted in a much greater retention of chromosome morphology thus facilitating direct comparisons of autoradiographs with photographs of banded spreads.

The recombinant RC8 contains a 5.1 kbp human insert and was nick-translated to a specific activity of 2.51 x 7 . . . 10 dpm/ug. The probe was hybridized overnight to slides at a concentration of lug/ml and exposed to Ilford L4 emulsion for 11 days at 4°C. Fig. 13 shows a spread with both X chromosomes labelled at Xp22 with a photograph of the banded chromosomes before hvbridization for

89 Fig.13 Human female metaphase spread A - after G- banding and B - after hybridization to RC8 and autoradiography. The two X chromosomes are labelled and in addition grains reside over chromosomes 7 and 22.

90 RC8 12 Female 6 Male Cells

Fig.14 Composite grain distribution over 18 cells after hybridization 'in situ' to RC8. Each dot represents the chromosomal position of a single grain. The banding pattern is from the Paris Conference, 1981 (ISCN, 1981).

91 Table 4

RC8 hybridised to 6 male and 12 female metaphases

Ch ro mo so me 0 E 0-E 1 3 8.49 - 5.49 2 10 8.28 1.72 3 2 6.85 - 4.85 4 13 6.55 6.45 5 7 6.28 0.72 6 3 5.92 - 2.92 7 7 5.43 1.57 8 2 5.00 - 3 .00 9 2 4.65 - 2.65 10 1 4.57 - 3.57 11 4 4.67 - 0.67 12 8 4.61 3.39 13 4 3.65 0.35 14 1 3.53 - 2.53 15 6 3.32 2.68 16 3 3.04 - 0.04 17 4 2.92 1 .08 18 3 2.75 0.25 19 3 2.12 0.88 20 1 2.35 -1.35 21 2 1.61 0.39 22 2 1.69 0.31 X 12 4.41 7.59 Y 0 0.30 - 0.30

S D = 3 .06817

The deviation of observed (0) from expected (E)

values (0-E) is estimated as a probability that the

deviation is random by the number of standard deviations

of all O-E values each 0-E value takes. Only in the case

of the X chromosome is the 0-E value greater than two

standard deviation. The probability that this value is

random is approximately 0.1%.

> 92 20-i XRC8 11 Chromosomes

pll

Fin.15 Distribution of grains over 11 X chromosomes

following 'in situ' hybridization to RC8 . The vertical

axis shows the composite number of grains. The horizontal

axis shows a G-banded X chromosome. The banding pattern is

from the Paris Conference, 1981 (ISCN, 1981). comparison. Fig. 14 is a diagrammatic representation of the composite number of grains over IB spreads and clearly demonstrates that this sequence is derived from Xp22. The

analysis of this result in Table 4 suggests only the X chromosome is significantly labelled (P<0.1%).

Further confirmation of this localization is accomplished by performing the hybridization at lQug/ml probe concentration. Under these conditions (with this probe), extremely high backgrounds are obtained and a full analysis becomes impossible due to large clusters of grains obscuring many chromosomes. The origin of these clusters is unknown, but presumably they represent extensive networks of radioactive probe. Fig. 15 is a diagrammatic representation of the composite grain di st r i b u t i o n over 11 X chromosomes v/hich remained unobscured by clusters. Of the total of 3.8 grains, 15 reside over Xp22.

Fig. 16 shows a metaphase spread after hybridization to the probe LI.28 and 20 days exposure to Ilford K2 emulsion at 4°C. The unhybridized banded spread is also shown for comparison. The probe contains a human DUA insert of 1.5kbp within the plasmid pBR322 (pavies et al.,

1983), was nick translated to a specific activity of 2.4 x - j 10 dpm/ug and hybridized at a concentration of lOOng/ml to microscope slides. The example autoradiograph) in Fig. 16 has one of the X chromosomes labelled at Xpl1. The composite grain distribution from 40 cells in Fig. 17 and the analysis in Table 5 provide convincing B

Fig.16 Human female metaphase spread A - after G- banding and B - after hybridization to LI.28 and autoradiography. One of the X chromosomes is labelled and in addition a single grain resides over chromosome 2.

95 LI.28 40 Cells

Fig.17 Composite grain distribution over 40 cells after hybridization to LI.28. Each dot represents the chromosomal position of a single grain. The banding pattern is from the Paris Conference, 1981 (ISCbT, 1981) .

96 Table 5

LI.28 hybridised to 40 female metaphases

Chromosome 0 E 0-E 1 9 10.66 - 1.66 2 8 10.39 - 2.39 3 9 8.59 0.41 4 6 8.22 - 2.22 5 10 7.88 2.12 6 8 7.44 0.56 7 6 6.82 - 0.82 8 4 6.28 - 2.28 9 10 5.84 4.16 10 5 5.74 - 0.74 11 7 5.86 1.14 12 7 5.79 1.21 13 5 4.58 0.42 14 1 4.43 - 3.43 15 6 4.16 1.84 16 3 3.82 - 0.82 17 0 3.67 - 3.67 18 2 3.45 - 1 .45 19 3 2.66 0.34 20 1 2.95 - 1 .95 21 0 2.02 - 2.02 22 0 2.12 - 2.12 X 20 6.65 13.35

S D = 3 .40881

The deviation of observed (O) from expected (E) values (0-E) is estimated as a probability that the deviation is random by the number of standard deviations of all 0-E values each 0-E value takes. Only in the case of the X chromosome is the 0-E value greater than one standard deviation. The probability that this value is random is approximately 0.006%.

97 evidence that this sequence is derived from proximal Xpl1

(P < 0 .0061).

5 2 a is a recombinant containing 1.7kbp of human DbA inserted into the plasmid pBR322 and is a subclone, freed of reiterated sequence, of a bacteriophage lambda recombinant isolated from the X-enriched library (Drayna et al., 1934). The recombinant was nich translated to a 7 specific activity of 6.74 x 10 dpm/ug and hybridized overnight to slides at a concentration of 100ng/ml. Fig.

13 is an autoradiograph after 20 days exposure at 4°C and one of the X chromosomes is labelled. The composite grain distribution over 40 cells is presented in Fig. 19. The seven grains over Xq27 suggest that 52A is derived from this region of the X chromosome but the statistical significance of this result is lower than other experiments (P<1.551, Table 6). This corresponds to a lower hybridization efficiency which may be due to diffusion constraints - the chromosome in this region might be highly condensed and consequently less accessible to probe. Alternatively the sequence might be more labile to fixation of cells in acid/methanol or the denaturing of the DdA. Small amounts of D'dA are lost from the chromosome during these treatments (Comings, 1978; Gall and Pardue,

1971 ) .

The recombinant S21 detects a locus in the region ql3 to qter as demonstrated in section 3.3. This was further localized by an 'in situ' hybridization experiment. The Fig. 18 Human female rnetaphase spread A - after G- banding and B - after hybridization to 52A. and autoradiography. One of the two X chromosomes is labelled and in addition grains reside over chromosomes 1, 6 and 7.

99 52 A 40 Cells

Fig.19 Composite grain distribution over 40 cells after hybridization 'in situ' to 52A. Each dot represents the chromosomal position of a single grain. The banding pattern is from the Paris Conference, 1981 (ISCO, 1981).

100 Table 6

52A hybridised to 40 female metaphases

Ch ro mo so me 0 E O-E 1 14 9.18 4.82 2 7 8.95 - 1.95 3 2 7.40 - 5.40 4 10 7.08 2.92 5 6 6.79 - 0.79 6 7 6.41 0.59 7 9 5.88 3.12 8 2 5.41 - 3.41 9 3 5.03 - 2.03 10 10 4.94 5.06 11 3 5.05 - 2.05 12 4 4.98 - 0.98 13 5 3.95 1.05 14 0 3.82 - 3.82 15 1 3.58 - 2.58 16 3 3.29 - 0.29 17 4 3.16 0.84 18 1 2.97 - 1 .97 19 2 2.29 - 0.29 20 2 2.55 - 0.55 21 1 1.74 - 0.74 22 4 1 .82 2.18 X 12 5.73 6.27

S D = 2 .90386

The deviation of observed (0) from expected (E) values (O-E) is estimated as a probability that the deviation is random by the number of standard deviations of all 0-E values each O-E value takes. The probability that the O-E value for the X chromosome is random is approximately 1.55%.

101 Fig.20 Human female metaphase spread A - after G- banding and B - after hybridization to S21 and autoradiography. Only the X chromosome is labelled with g r a i n s .

102 at rn s rm h Prs ofrne 13 (SN 1981). (ISCN, 1931 Conference, Paris represents the dot from Each is n . er 1 tt 2 pa S to situ' 'in n io at iz id br hy after the chromosomal po si ti on of a single grain. The banding banding The grain. single a of on ti si po chromosomal the

F i g .21 Composite grain di st ri bu ti on over 36 cells cells 36 over on ti bu ri st di grain Composite .21 g i F ai ■ i iBrnm S2I 36 Cells 36 103 Table 7

S21 hybridised to 36 female metaphases

Chromosome 0 E 0-E 1 2 6.48 - 4.48 2 6 6.31 - 0.31 3 5 5.22 - 0.22 4 3 5.00 - 2.00 5 3 4.79 - 1 .79 6 1 4.52 - 3.52 7 6 4.14 1 .86 8 4 3.82 0.18 9 3 3.55 - 0.55 10 4 3.49 0.51 11 3 3.56 - 0.56 12 1 3.52 - 2.52 13 1 2.78 - 1.78 14 1 2.69 - 1.69 15 1 2.53 - 1 . 5 3 16 3 2.32 0.68 17 3 2.23 0.77 18 2 2.09 - 0.09 19 0 1.62 - 1 .62 20 3 1 .80 1.20 21 1 1.23 - 0.23 22 3 1.29 1.71 X 20 4.04 15.96

S D = 3 .73982

The deviation of observed (0) from expected (E) values (0-E) is estimated as a probability that the deviation is random by the number of standard deviations of all 0-E values each 0-E value takes. Only in the case of the X chromosome is the 0-E value greater than one standard deviation. The probability that this value is random is approximately 0.001%.

104 DMA was nick translated to a specific activity of 4.94 x 7 10 dpin/ug and hybridized at a concentration of lOOng/ml to

slides which were exposed to Kodak NTB-2 emulsion for 22

days at 4°C. Fig. 20 shows the resultant autoradiograph

with one X chromosome labelled and in Fig. 21 the

composite grain distribution over 36 spreads is displayed

diagramatically. The only chromosome with a significant

number of grains covering it is the X and clearly S21 is derived from a sequence at Xq22-q23 (P<0.001%, Table 7).

A recombinant has been reported which detects homologous loci on both the X and Y chromosomes (Page et

al., 1932). This recombinant, DP31, contains 4.5kbp of human DMA cloned into pBR322. The X chromosome locus has been investigated by 'in situ1 hybridization in this

study. The probe was nick translated to a specific

activity of 2.62 x 10 dpm/ug and hybridized at a

concentration of lOOng/ml to slides which were exposed to

Kodak MTB-2 emulsion for 22 days. Figs. 22-23 represent

the result. Fig. 22 is a hybridized spread showing one of

the X chromosomes labelled at Xql3 and Fig. 23 represents

the composite grain distribution over 37 spreads. The X chromosome remains the only significantly labelled

chromosome (female cells were used and hence the Y locus was not investigated) and it seems unequivocal that the X

locus is at Xql3 (P<0.009%, Table 0).

The paradox of when a single-copy sequence is repeated is raised by the recombinant DX13. This lambda

recombinant contains two human EcoRI fragments of 2.2 and

105 Fig.22 Human female metaphase spread A - after G- banding and B - after hybridization to DP31 and autoradiography. One of the two X chromosomes is labelled and in addition a single grain resides over chromosome 1.

106 DP3I 37 Cells

Fig.23 Composite grain distribution over 37 cells after hybridization 'in situ' to DP31. Each dot represents the chromosomal position of a single grain. The banding pattern is from the Paris Conference, 1981 (ISCN, 1981).

107 Table 8

DP31 hybridised to 37 female metaphases

Chromosome 0 E 0-E 1 12 11 .89 0.11 2 7 11.59 - 4.59 3 15 9.58 5.42 4 8 9.17 - 1.17 5 15 8.79 6.21 6 4 8.29 - 4.29 7 10 7.61 2.39 8 4 7.00 - 3.00 9 6 6.51 - 0.51 10 6 6.40 - 0.40 11 4 6.54 - 2.54 12 11 6.45 4.55 13 2 5.11 - 3.11 14 5 4.94 0.06 15 4 4.64 - 0.64 16 4 4.26 - 0.26 17 2 4.09 - 2.09 18 0 3.84 - 3.84 19 0 2.97 - 2.97 20 0 3.30 - 3.30 21 1 2.25 - 1.25 22 0 2.36 - 2.36 X 25 7.41 17.59

S D = 4 .73838

The deviation of observed (0) from expected (E) values (0-E) is estimated as a probability that the deviation is random by the number of standard deviations of all 0-E values each 0-E value takes. Only in the case of the X chromosome is the 0-E value greater than two standard deviations. The probability that this value is random is approximately 0.009%.

108 Fig.24 Human female metaphase spread A - after G- banding and B - after hybridization to DX13 and autoradiography. The two X chromosomes are labelled and in addition grains reside over chromosome 7.

109 DXI3 14 Cells

1 1

’ : ¥ 7 R ' f S i qi: ‘■y-y. ) ■.i ___

I6 I7 I8 19 20 21 22 Y

Fig.25 Composite grain distribution over 14 cells after hybridization 'in situ' to DX13. Each dot represents the chromosomal position of a single grain. The banding pattern is from Paris Conference, 1981 (ISCN, 1931).

110 Table 9

DX13 hybridised to 14 female metaphases

C h ro mo so me 0 E 0-E 1 6 6.23 - 0.23 2 0 6.07 - 6.07 3 6 5.02 0.98 4 1 4.81 - 3.81 5 3 4.61 - 1.61 6 4 4.35 - 0.35 7 4 3.99 0.01 8 6 3.67 2.33 9 4 3.41 0.59 10 6 3.35 2.65 11 2 3.43 - 1.43 12 2 3.38 - 1 . 3 8 13 2 2.68 - 0.68 14 2 2.59 - 0.59 15 2 2.43 - 0.43 16 3 2.23 0.77 17 1 2.14 - 1 . 1 4 18 2 2.02 - 0.02 19 2 1.55 0.45 20 0 1.73 - 1.73 21 0 1.18 - 1 . 1 8 22 0 1.24 - 1 . 2 4 X 18 3.89 14.11

S D = 3 .48924

The deviation of observed (0) from expected (E) values (0-E) is estimated as a probability that the deviation is random by the number of standard deviations of all 0-E values each 0-E value takes. Only in the case of the X chromosome is the 0-E value greater than one standard deviation. The probability that this value is random is approximately 0.001%.

I l l l.Okbp yet hybridizes to five human EcoRI fragments of

1.3, 2.2, 2.5, 2.7 and 7.4kbp. All four of these fragments

co-segregate with the human chromosome in IIorl9X, IUI-5

and MCP-6 (results not shown) and so all must be derived

from Xql3-qter. This evidence is suggestive of a localized

repeated sequence and further corroboration is provided by

an 'in situ' hybridization experiment. The recombinant was

hybridized to slides overnight at a concentration of

500ng/ml and a specific activity of 2.35 x 10 dpm/ug.

Slides were exposed to Ilford K2 emulsion for 25 days at

4°C and Fig. 24 is an autoradiograph depicting label on

both X chromosomes at Xq23. In Fig.25 the genomic

distribution of this repeated sequence is revealed and

clearly it is derived from a single chromosomal locus at

Xq28 (P<0.001%, Table 9). Such a discrete block of

homologous sequences is reminiscent of many chromosomal

loci including that of the histone genes (Heintz et al.,

1931).

C Summary of 'in situ' hybridization data

The chromosomal loci of six human X chromosome DMA

sequences have been determined to a high resolution by 'in

situ' hybridization. The composite X chromosomal grain distribution of these 'in situ' hybridization experiments

is presented schematically in Fig. 26. The height of each bar represents the composite number of grains over each

band from all the chromosomes scored. The specificity of

the technique is evident from this diagram and clearly the

112 2

XS2I 72 Chromosomes

20i p52A XDXI3 \ 80 Chromosomes 50 Chromosomes s \s s s\ k> io- s s s \ \

q28

Fig.26 Composite X chromosomal grain distributions.

113 Fig.27 Cytological map of the human X chromosome from

'in situ' data. The locus each marKer detects is taken as the G-band or interband with the greatest total number of grains from each experiment. The banding pattern is that from the Paris Conference, 1981 (ISCN, 1981).

114 six recombinants detect discrete loci spread along the chromosome from Xp2 2 ( RCR) to Xg20 (DX13). This then provides the basis for the cytological nap of the human X chromosome displayed in Fig. 27. The map reported here agrees with the chromosomal localization of many of the same probes using somatic cell hybrids (L'ieacker et al.,

1983; see section 3.3.).

The particular relevance of these six DMA probes is that in addition to detecting discrete cytological loci, they each detect restriction fragment length variants.

These variant fragments act as inheritable markers conseguentlv permitting linkage distances between each pair of loci to be computed.

3.5. Restriction fragment length polymorphism

A RFLP and heterozygote detection

The use of restriction length variants in gene mapping and linkage analysis has become well established and is the subject of many recent reviews (Kurnit and lloehn, 1979; Botstein et al., 1980; Davies, 1981). The identification of RFLP has been approached in different ways. The stochastic approach is to screen a large number of random female DMAs digested v/ith different restriction enzymes v/ith a single probe. Such a screen has revealed

RFLP detected by RC8 (Murray et al., 1982), by LI.28

(Davies et al., 1983) and by S21, 52A and DX13 (Drayna et

115 al., 1934). An internediate approach is to screen a snail

number of male and female DMAs digested with many enzymes

as has been successfully applied for DP31 (Page et al.,

1932) or indeed to screen just two females and one male

with very many enzymes. This latter approach offers the

advantage that only very frequent X-linked polymorphism

will be detected and it is this very frequent polymorphism which lends itself most amenable to linkage analysis.

An example of the variants detected by the

recombinant RC3 is provided in Pig. 23. Female DPAs ''/ere

digested with the restriction endonuclease Tag I, blotted . . 32 onto nitrocellulose and nyondized to p nick translated

HC3. The autoradiograph demonstrates that this recombinant

hybridizes to three TaqI fragments of molecular weights

6.6, 5.3 and 3.2kbp. The largest of these fragments is

invariant in all individuals tested but the 5.3kbp band

interchanges with the 3.2kbp band. Pales disolay only the

5.3kbp band or the 3.2kbn band and never both (hurray,

1934). Consequently it appears that females with. both bands are heterozygous for the mutation leading to the absence (or creation) of a TaqI site at this chromosoma1 locus and can give birth to male offspring inheriting either fragment. This conclusion has been confirmed by exhaustive inheritance studies of these restriction

fragments (Murray, 1934)*

Genetic mappingof the X chromosome is greatly facilitated by the fact that the male is effectively

116 1 2 3 4

Fig.28 TaqI fragment variants detected by RC8. Lanes

1 and 2 are female blood DMAs digested to completion with

TaqI. Lanes 3 and 4 are male blood DMAs.

117 haploid for the X and consequently there is no disruption

through recombination in the chromosomal order of X-linked

alleles in father to daughter transmission. Consequently

once the restriction fragment variants of a father's X

chromosome has been determined the linkage phase of his

daughter's X chromosome can be absolutely defined. This

permits the accurate definition of sites of crossover in

offspring when the RFLV constitution of the grandfather,

mother and father are known.

Some three generation pedigrees were available for

this study in the form of lvmphoblastoid cell lines

derived from leukocytes or as short term peripheral blood

cultures stimulated with a mitogen. The first step of

linkage analysis in such pedigrees is the identification

of mothers heterozygous for mutations leading to RFLP.

Consequently DNA from each mother was digested with

various restriction enzymes, fractionated by agarose gel

electrophoresis and transferred to nitrocellulose. Filters were hybridized with the six recombinants known to detect

RFLP and also with recombinants which had not been

screened for RFLP. The advantage of using the mothers to

screen for RFLP is that any heterozygotes identified can

instantly be used in linkage analysis. However, no RFLP was detected for the recombinant DX4 with DMAs digested

with either TaqI, FspI, Bglll or SstI (results not shown).

Fig. 29. shows the result of hybridizing the

recombinant S21 to TaqI digested DHAs of seven of the

eight mothers screened in this study. This 7.3kbp sequence

118 12 3 4 5 6 7 8

Fig.29 TaqI fragment variants detected by S21. Each

lane represents female cell line DNA digested to completion with TaqI. Lane 1 is molecular weight markers of 23.1, 9.4, 6.6 and 4.4kbp; Lane 2 - D3II2; Lane 3 -

D 2112 ,* Lane 4 - D2II? ; Lane 5 - BD26II2; Lane 6 - BD26II4 ;

Lane 7 - DlII^; Lane 8 - DlII^.

119 hybridizes to two invariant bands of 4kbp and 2.5kbp and

detects variant bands of 3.2kbp and 3kbp. Hales inherit

only the 3.2kbp or the 3kbp fragment and never both.

Consequently, heterozygotes display both fragments and

homozygotes only one. In the figure, females D2Il2»

D2II_, and BD26II^ are homozygous for the commoner 3.2kbp

fragment (p=0.65) and female DlII^ homozygous for the

rarer 3kbp (q= 0.35)• Obviously genetic exchange between

the two X chromosomes will not be detected in these

mothers but females ED26II_ and Dill,, can pass on either 2 o fragment to their offspring and hence recombinants between

this locus (q22-23) and an adjacent locus readily

identi f ie d .

The DMA constitution at six loci was determined for

the eight mothers screened in this study. Table 10

displays the results of these experiments. The six loci

are represented by the recombinants which detect both

their cytological location and genetic constitution and

are arranged in the order in which they are located on the

chromosome from RC3 at Xp22 to DX13 at Xq23 (see Fig. 27).

Mothers who are heterozygous at adjacent loci are clearly defined and by studying inheritance of restriction

fragments from these mothers, recombinants between two cytological loci can be determined. For example, the two sisters, females 0311^ and D3II are heterozygous at the

L.1.28 and DP31 loci. Thus, once the phase of their chromosomes has been determined (by determining the

120 Table 10

Female Locus

RC8 LI .28 DP31 S21 52A D X 1 3

D1 II4 Bl/Bl Cl/Cl E2/E2 J2/J2 Ll/Ll Ml /Ml

Dl II Bl/Bl Cl/Cl E2/E2 J2/J1 Ll/Ll Ml/Ml 6 D 2 I I ? B2/B1 C1/C2 E3/E3 Jl/Jl L2/L1 M2 / M2

D 2 1 1 ? B2/B1 C1/C2 E3/E3 Jl/Jl L2/L2 M2/M1

Bl/Bl C2/C1 E3/E2 Jl/Jl L2/L1 Ml/Ml D 3 I I 2 Bl/Bl C2/C1 E3/E2 Jl/Jl Ll/Ll Ml/Ml D 3 I I 4 BD26II2 Bl/Bl C2/C1 E2/E3 J2/J1 L2/L1 M1/M2

BD2611 . Bl/Bl C2/C1 E3/E3 Jl/Jl Ll/Ll M l / M2 4

DNA sequence variants at six X chromosome loci in eight mothers from three generation pedigrees (Dl, D2, D3 and ED26)

RC8 TaqI fragments B1 3.2kbp, B2 5.3kbp

LI.23 TaqI fragments Cl 12kbp, C2 9kbp

DP31 TaqI fragments El 14.6kbp, E2 ll.Bkbp, E3 lO.Gkbp

S21 TaqI fragments J1 3.2kbp, J2 3kbp

52A TaqI fragments LI 0.6, 0.7kbp, L2 1.3kbp

DX13 Bglll fragments Ml 2.8kbp, M2 5.8kbp

121 fragments on their father's X chromosome), recombinants

between these two loci can be detected in their offspring.

B Inheritance studies

Having identified informative mothers in the three

generation pedigrees available, DUA was prepared from all

the family members, digested with the appropriate

restriction endonuclease (TaqI for probes RCS, LI.28,

DP31, S21 and 52A; EglII for probe DX13) and blotted to

nitrocellulose. Filters were hybridized to radioactively

labelled recombinant probes and hybridized fragments

detected by autoradiography. Fig. 30 shows the result of

screening pedigree D3 with the probes LI.23 and DP31.

LI.23 detects up to two Tag I fragments of 12kbp and 9kbp

in females - only one of these fragments is found in males

(Davies et al., 1983). The inheritance of these fragments

is revealed in the figure - the grandfather (D3I ) has

transmitted the 9kbp fragment (C2) to the mothers (D3II?

and D3II^) who have inherited the 12kbp alternative (Cl)

from the grandmother (D3I^). Consequently the offspring in

the third generation can inherit either fragment from

their mother - the departure of expected Mendelian ratios of 50% of the offspring inheriting each fragment is not

significant in this small a sample.

The pattern revealed when DP31 is hybridized to a

TaqI digest of pedigree D3 is more complex (Fig. 30B).

This recombinant detects a 14.6kbp TaqI fragment of the Y chromosome and either an ll.Bkbp or a lO.Gkbp fragment of

122 A

Fig.30 TaqI fragment variants in pedigree D3 detected by A - LI.28, B - DP31. Lane 1-1 , Lane 2-I2# Lane 3-II ,

Lane 4-111-^, Lane 5-III2, Lane 6-111^, Lane 7-II , Lane 8-

II4 , Lane 9-III , Lane 10-III , Lane ll-III .

123 Fig.31 Pedigree D3 showing the inheritance of Tagl variants detected by LI.28 (Cl and C2) and by DP31 (El , E2 and E3) with Duchenne muscular dystrophy. The El var iant is linked to the Y chromosome. Affected offspring are shaded, carriers are marked with a dot.

124 the X. The inheritance of the Y fragment (El) is shown by the transmission from the father II„ to his sons III and S III . The mothers (II0 and 11^) have inherited the ll.Ghbp fragment (E2) from the grandmother I and the lO.Ghbp fragment (E3) from the grandfather I and transmit either

X-linked sequence to the third generation. As the X chromosome in I has the fragment C2 at the LI.28 locus and the fragment C3 at the DP31 locus, the phase in both mothers must be C2E3/C1E2. Consecuently, any sons with the chromosomes C2E2 or C1E3 nust be recombinants and those with C2E3 or C1E2 are non-recombinants. The pedigree in

Fig. 31 shows that one of the two sons in each family

(III and II lr) are recombinants. In addition, III has inherited the C1E2 chromosome from her father II and is also a recombinant having therefore inherited a C2E2 chromosome from her mother. Ill may or maynot be a recombinant depending on her father's X chromosome - if he were C1E3 then she is not a recombinant whereas if he were

C1E2 then she must hie a recombinant. Unfortunately the father was not available for testing. The pedigree D3 also segregates for Duchenne muscular dystrophy as shown. The segregation of the DXA at the LI.28 locus with the

Duchenne locus is commensurate with the 17cli genetic distance established between the two (Davies et al. ,

1933).

The RULE for the LI.28 locus is also demonstrated in the pedigree D2. In addition the RFLP detected by RCO is

125 Fig.32 Pedigree D2 showing the inheritance of TaqI fragments detected by LI.28 and RC8 with Duchenne muscular dystrophy. The TaqI variants detected by LI.28 are termed

Cl and C2 and by RC8 are termed B1 and B2. Affected offspring are shaded, carriers are marked with a dot.

126 also evident in this pedigree. Fig. 32 shows how the 12kbp

(Cl) and 9kbp (C2) Taol fragments detected by LI. 23 segregate with the 3.2kbp (P.l) and 5.3kbp (132) Tacrl fragments detected by RGB. The grandfather's (1^) chromosome is B2C1 thus the phase in both mothers is

B2C1/B1C2 and recombinants in the third generation will be

B2C2 or B1C1. There are no recombinants in the offspring of IIn (the daughter II^ is homozygous at both loci and thus has inherited trie non-recombinant B1C2 chromosome from the mother) and a single recombinant in the offspring of II_ - III-, who has inherited the B1C2 chromosome from 7 3 her father and therefore the B1C1 recombinant chromosome from her mother. The diagnostic potential of these RFLP markers is revealed by following their segregation with the Duchenne phenotype in this pedigree. The mutant chromosome also carries the D1C2 Tagl fragments in the mothers and the two affected offspring. This observation taken with the linkage data available between DC 8 and DTP and LI.28 and DFD (Davies et al., 1933), allows one to predict that III9 and III7 who also have the R1C2 chromosome from their mothers, will be carriers for DI .D.

Table 10 shows that mother DD2GII^ is heterozygous at each of the six loci investigated bar that detected by

RC3 . The TaqI fragments detected by DP31 and the Bglll fragments detected by DX13 in the individuals from the pedigree are shown in Fig. 33. DX13 detects six Bglll fragments, four invariant bands of 8.3kbp, 1.7kbp, 1.4kbp and 0.9kbp and two variant bands of 5.Bkbp and 2.Bkbp.

127 12 3 4 5 6 7

B

Fig.33 Restriction fragment variants in pedigree

BD26. A TaqI fragments detected by DP31. B Bglll fragments detected by DX13. Lane 1-1^, Lane 2-I2, Lane 3-II2, Lane

4 -H 4 1 , Lane 5-111^, Lane 6 -III3# Lane 7-III4 .

128 U u in -J ^

Fig.34 Pedigree BD26 showing the inheritance of restriction fragments detected by LI.28, DP31, S21, 52A and DX13. Variants are termed for; L1.28-C1,C2; DP31-

E 2 , E 3 ; S21-J 1 ,J 2 ; 52A-L1,L2; DX13-M1,M2.

129 Only one of the variant bands is ever seen in males. The inheritance of these Bglu fragments along with the TaqI fragments which hybridize to LI.28, DP31, S21 and 52A is

shown in Fig. 34. Unfortunately the mother who is heterozygous at all these loci has only a single son but remarkably no recombination can be detected in this son's

X chromosome from the LI.23 locus (Xpll) to the DX13 locus

(Xq28). The grandfather's chromosome is C1E3J1L1M2 and the mother, II , therefore has the phase ClE3J1L1M2/C2E2J2L2H1 and her son (III ) has inherited the non-recombinant chromosome C2E2J2L2M1. It seems quite extraordinary for there to have been no recombination in the interval Xpll- Xcen-Xqter at a single meiosis and it seems more likely that two crossovers have occurred between adjacent loci.

As chiasma interference does not extend across the centromere in humans (ilulten, 1974) and crossover interference does not extend across the centromere in Drosophila (Muller, 191G), the most likely site of a double crossover is between the loci detected by DP31

(Xql3) and LI.28 (Xpl1).

3.6. Genetic distances a genetic map of the X

The paucity of informative meioses available for this study prohibit a high lod score for the maximum likelihood estimate of theta between any two loci. However, Table 11

130 Table 11

A - Li.28 v DP-31

Mother No. Recombinants Recombination Traction (theta)

0.01 0.05 0.] 0.15 0.2 0.25 0.3 0.35 0.4 0.45

D3II.2 1/2 -1.402 -0.721 -0.444 -0.292 -0.194 -0.125 -0.076 -0.041 -0.018 -0.004

D3II.4 2/3 -3.101 -1.721 -1.143 -0.815 -0.592 -0.426 -0.298 -0.196 -0.115 -0.050

BD26II.2 0/1 0.297 0.279 0.255 0.230 0.204 0.176 0.146 0.114 0.079 0.041

TOTALS -4.206 -2.163 -1.332 -0.877 -0.582 -0.375 -0.228 -0.158 -0.054 -0.013

B - RC8 v LI.28

Mother No, Recombinants Recombination Fraction (theta)

0.01 0.05 0.1 0.15 0.2 0.25 0.3 0.35 0.4 0.45

D2II.2 0/2 0.593 0.553 0.511 0.461 0.403 0.352 0.292 0.228 0.158 0.083

B2II.7 1/3 -1.106 -0,442 -0.188 -0.062 0.010 0.051 0.070 0.073 0.061 0.037

TOTALS -0.513 0.116 0.323 0.399 0.418 0.403 0.362 0.301 0.219 0.120

Lod scores from the inheritance studies in this work. The Lod values from each pedigree were obtained from

standard tables or using the computer program LIPED.

131 shows the loci scores at different values of theta between the loci detected by DP31 and LI.28 (derived from pedigrees D3 and BD26) and between those detected by LI.20

and RC8 (derived from pedigree D2). From this data i t can be seen that linkage is suggestive betv/een RCS and LI .28 Q t a recombination fraction of 0.2-0.25 and unlikely

betv/een LI.28 and DP31 The sequential method of determining the maximum likelihood estimate permits this data to be combined with

similar analyses elsewhere and a more unequivocal estimate achieved. Ouch an analysis has been performed by Drayna in Salt Lake City and the combined data suggest the following

genetic distances where Xcj is the locus for the X-l inked

blood antigen at Xp223 (Ferguson-Smith et al., 1902).

Xg - RCS > 50cM RC8 - LI.20 3 3 cM

LI.28 - DP31 > 50c M

DP31 - S21 14 c H S21 - 5 2A 4 5cli 52A - DX13 4Gci 1 Th ese geneti distances are still equivocal (Drayna et al., 1904) but comparison with the cyto logical map of Fig. 27 is possibl e. Fig. 35 represents this schematically by interpreting d ifferences between genetic and physical

distances as an expansion or contraction of the mitotic

metaphase G-bands. The physical locus is taken as the mid- point of the band each marker has been assigned to by 'in

situ' hybridizati on. The genetic locus is taken from the

132 f Xg

Physical Genetic

Fig.35 Physical and genetic maps of the human X chromosome. The relative position of band-interband boundaries to flanking loci is converted to a fraction of the genetic distance separating the loci to generate the genetical representation of the chromosome shown.

133 genetic map of Drayna et al (1984) Clearly the genetic distances between the RC3 locus and the qene for the Xq surface antigen at Xp223 (Ferguson-Smith et al. , 1932) and between the loci for 52A (Xg27) and DX13 (Xq23 ) grossly differ from the comparative length of the metaphase chromos'orne that separates them. This impl ies that recombination is occurring far more f requently in proterminal regions.

134 CHAPTER 4 Discuss ion

135 4.1. DMA seq uence organization of the human X

chromosome

The results from this study demonstrate the distribution of some repetitive DMA sequences over the X chromosome. The observation that total MCP-S DMA reacts with the same recombinant sequences as total Horl9X DMA suggests that there are no repetitive sequences on the X chromosome Inching in the translocation chromosome t(X;6)(Gqter-6p21::Xql3-qter). This therefore implies that there are no centromeric repetitive sequences specific to the X. However a 2kbp Bamlll repeat sequence has been reported to be highly represented at Xcen (Yang et al.,

1902; Millard et al., 1923) and 'in situ* hybridization with this sequence reveals grains almost exclusively at

Xcen (Yang et al., 1932; Cates and Hartley, unpublished). The conclusion must be that this repeated sequence was not represented in any of the recombinants probed '-4th somatic cell hybrid DMA. This conclusion is strengthened by analysis of the repeated sequence. Millard et al. (1933) report that the 2kbp repeat is predominantly tandemly repeated at a pericentromeric location on the X chromosome. Southern blot analysis to total EcoRI digests of human DMA indicated that this enzyme primarily cleaves outside of the tandem repeat and consequently most EcoRI fragments containing this repeat migrate as high, molecular weight DMA. As a consequence even though this sequence may make up to as much as 3-103 of X chromosomal DMA it would

136 be expected to make up a significantly smaller proportion of EcoRI digested DMA in the size fragment range 2-14 kbp.

This is the target DMA capacity of X gtt'ES "Xb and consequently the expected insert size range in the X- enriched library (and indeed the largest insert size observed was 7.9kbp). As only 1000 or so recombinants were analysed in this comparative screen, it appears likely that the repeat makes up less than one per cent of the cloned sequences present in the library and therefore of the order of one per cent of X chromosomal DMA EcoP.I fragments in the fragment size range 2-14kbp (the library is not completely representative of X chromosome sequences and so this will additionally dilute the BamHI repeat). ' 'hen cloned repetitive DMA sequence probes were used in 'in situ' hybridization experiments, two types of distribution were observed . The Hindi 11 and Alu re'peats are apparently dispersed randomly along the chromosome whilst the ocRI and BamHI repeats are clustered. It is hard to see how the clustered repeats are involved in gene regulation as postulated by Davidson and Britten (1973) but perhaps their centromeric location, similar to the distribution of human satellite DMA sequences (Mosden et al . , 1975 ), indicates a functional role in chromosome identification and segregation at mitosis and meiosis. The sequence conservation of primate alnhoid DMA sequences such as the EcoRI 340bp dimer, the African green monkey component alpha DMA (Manuelidis and Hu, 1978) and the

137 BamHI 2kbp repeat (Yanget al., 1982) as v/ell as their similar chromosomal organization agrees with this hypothes i s.

It has been proposed that repeated DMA. sequences occupy different concentrations in Giemsa-dark (G) and

Giemsa-1ight (R) bands (Comings, 1979). However, the apparently random distribution of flindlll and Alu sequences argue against this hypothesis. In addition, comparative analysis, of G and R band DMA from Chinese hamster cells demonstrates no differences in their rate of reassociation (Holmquist et al., 1982). The dispersed distribution of HindiII and Alu repeats provides circumstantial evidence for uniform condensation along the chromosome. If it is assumed that the periodicity of interspersion of these repeated sequences is relatively constant, as seems to be the case for intermediate repetitive sequences in general (Schmid and Deininger, 1975), then their uniform distribution implies a uniform distribution of DHA exhibiting this type of interspersion along the metaphase . chromosome. This conclusion must be taken with the caveat that a significant percentage of non-repetitive human D8A occurs in stretches of greater that 20kbp (Schmid and Deininger,

1975). In addition, these non-renetitive sequences are generally absent from highly repetitious 'satellite' !)HA which remains highly condensed throughout the cell cycle (Brutlag, 1980).

138 4.2. The construction of chromosome maps in mammals

Considerable attention has been devoted recently to the new methodologies in mammalian gene mapping (Ruddle, 1431). In this study both somatic cell genetics and 'in situ' hybridization have been used in the construction of a cytological map of the human X chromosome. Each of these techniques suffers from disadvantages. The production of the human x rodent hybrid cells retaining specific human chromosomes presents difficulties in the absence of a selective marker on the chromosome such as HGPRT on the X, thymidine kinase on 17 or adenine phosphoribosyltransferase (APRT) on 16. Chromosomes which are not retained by selection are subject to continual loss during growth thus generating considerable mosaicism which must be monitored by enzyme analysis and karyotyping for accurate DHA sequence mapping. In addition, some somatic cell hybrids can be labile to chromosome rearrangement during growth which nay be beyond the resolving power of the microscope. The main drawback to the use of somatic cell hybrids in intra-chromosomal gene map-ping is the relative paucity of derivative chromosomes such as translocations or deletions. This can be circumvented in part by constructing deleted chromosomes through radiation-induced chromosome breakage (Goss and Harris, 1975). However, the characterization of such

139 fragments by karyotyping is a formidable task and their mitotic stability is poor. The most favourable aspect of

somatic cell genetics is the relative ease of chromosomal

assignment of a gene or DbA segment once suitable hybrids

have been constructed, either by Southern blot analysis or by antigenic expression or enzyme assay. Thus for

chromosomal assignment the use of somatic cell hybrids

remains particularly powerful, but in the absence of a large number of well characterized derivative chromosomes its resolution remains inadequate for the construction of cytological maps.

The resolution afforded by 'in situ' hybridization outstretches that of somatic cell genetics and as this work demonstrates, permits a number of discrete loci to be mapped onto a single chromosome to the level whereby genetic distances can be estimated and compared to their physical counterparts. This resolution coupled with the

use of normal human chromosomes in a normal cellular environment as target are the great advantages this

technique bestows upon gene mapping. The main disadvantages are the technical difficulty of the experiment and the time taken for autoradiography. The technical difficulties are surmounted by good cytogenetic

practice and careful probe preparation. Autoradiographic exposure times can be reduced by the use of different 1 ? 5 35 radioactive isotopes such as “ I or 5. Iodine-125 suffers from a much lower resolving power than trittium (Ada et al., 1956) and Sulphur-35 as a higher energy beta

140 emitter causes the production of more than one grain per

disintegration, thus again reducing resolution. DLTA probes

v/hich are detected by non-isotopic means offer particular

amelioration to this technique (Langer-Safer et al., 1932)

provided trie sensitivity rivals that of radioisotopic label.

The analysis of autoradiographs after hybridization represents the most subjective element to 'in situ' hybridization. This subjective element was minimized in this study in three ways. Firstly slides were banded and photographed before hybridization for unequivocal karyotyping and ensuring a random collection of hybridized spreads. Secondly autoradiographs were scored blind (i.e. ignorant of the probe) and more than one probe was

hybridized in each experiment (to different slides) to minimize bias on scoring grains. Finally the statistical significance of each result was estimated by comparing the

observed distribution of grains to a purely random distribution. In this fashion, a high resolution of mapping was achieved, relatively free from bias and

specific to the DPT probe.

The six cytogenetic loci determined in this study represent a significant contribution to a completely

mapped human X chromosome. T precise human genetic map must be a prerequisite for comprehending’ each gene's function and mode of expression and thus defining all aspects of normal and abnormal human biology and

141 development. There are over one hundred X-linked Mendelian

traits in man (McKusick, 1983) whose linkage to any one or more of these six loci open the way to genetic counselling and prenatal diagnosis. The deficiencies associated with the alpha and beta globin gene clusters (beatheral1 and

Clegg., 1982) have become the classical example of the use of linked DU A sequence variants in prenatal diagnosis. Restriction fragment length variants have been reported assoc^ited with mutant genes (Little et al., 1980; Kan and

Dozy., 1978) or caused by the mutation giving rise to a mutant phenotype itself (Geever et al., 1981). Two of the loci mapped in this study have proved useful in the mapping of the loci for Duchenne (Murray et al., 1982; Davies et al., 1983) and Becker (Kingston et al., 1983) muscular dystrophies and also in determining the carrier status of females in affected families (Penbrey et al., 1933). Using a cloned fragment of the HGPRT gene (Jolly et al., 1932) to detect BamHI restriction fragment variants and a cloned fragment of the gene for blood clotting

factor IX (jaye et al., 1983), the loci detected by 52A and DX13 are shown to lie in the genetic order HGPRT-52A-

FIX-PX13 relative to these genes (Dravna et al., 1934). Similarly there is equivocal data on the linkage between the locus detected by 52A and the inheritance of X-linked mental retardation associated with a fragile site at Xq27- 28 (Tariveruian and T’eck, 1982) given that the FIX locus is linked to this locus (Camerino et al., 1933).

Although individually the loci mapped in this study

142 may be of limited diagnostic potential for X-linked disorders, the value of such randomly isolated genomic sequences in gene mapping and diagnosis has been made dramatically clear in the case of Huntingdon's chorea (Gusella et a1., 1983).

4.3. Localized recombination

A comparison of estimated physical distances between chromosomal loci and their albeit equivocally determined genetic counterparts indicates clear differences in the case of the human X chromosome. The diagrammatical comparison in Fig. 35 indicates that recombination occurs much more frequently than expected given physical distance in proterminal regions and additionally in the vicinity of the centromere on the long arm. The primary caveat to this conclusion must be the equivocal genetic distances in some cases. In addition the assumption is that the condensation of the mitotic metaphase and meiotic pachytene DMA along the chromosome length is uniform. This assumption seems valid from electron microscopical analysis of chromosomes

(DuPraw, 1970; Miller and Hamkalo, 1972) and from the uniform distribution of DMA specific stains along the chromosome (Carrano et al., 1979). The equivalence of the mitotic metaphase and meiotic pachytene chromosome seems reasonable from measurements of relative lengths and

143 centromeric indices, as well as banding patterns (Ilulten, 1974; Holm and Rasmussen, 1977).

Genetic maps derived from measurements of chiasma distribution have been compiled only for chromosomes 1, 2 and 9 (tiulten et al., 1982, Laurie et al., 1982). The similarity between genetic maps prepared in that fashion with the map of the X in this work is convincing evidence for Janssens' chiasmatype theory. For example, the diagrammatic representation of chromosome 9 in Laurie et al. (1932) bears a striking resemblance to Fig. 35 representing the genetic map of the similarly sized X chromosome.

The implication of localized recombination as evinced both by this work and experiments demonstrating chiasma localization is for differential DMA sequence affinity for recombination along the human chromosome's length. This conforms with chiasma localization observed in piany species, including the mouse (Polani, 1972), newts (Callan and Perry, 1977), locusts (Fox, 1973), grasshoppers (Shaw,

1974) and higher plants (Rees and Dale, 1974) and with crossing over localization in Drosophila (Schalet and

Lefevre, 1971). This observation is also made at the DMA sequence level when large blocks of cloned genomic sequence are examined. Thus in a region of the short arm of chromosome 11 adjacent to the insulin gene, Lebo et al

(1933) found that a tandemly repeated sequence was apparently labile to recombination in a region which exhibited overall less recombination than exoected based.

144 on physical lengths. In the immune response region from the murine major histocompatibility complex on chromosome

17/ all recombination between the I-A and I-E subregions

(0. lcn) maps to a region of DMA of at most 9. Okbp

(Steinmetz et al., 1982). If one extends the analogy of genetic domains in this chromosome to the human X, the 233cM chromosome would consist of 2,400 of such domains. As the X consists of approximately 1.3 x 10 kbp

(Mendelsohn et al., 1973), each genetic domain would therefore correspond to approximately 75kbp, a figure which corresponds remarkably to the size of chromosome domains detected by microscopical and biochemical techniques.

The non-random distribution of cross-over sites and chiasmata in Drosophila prompted Mather (1938) to propose that chiasma were formed sequentially. By his model, the first chiasma in the Drosophila oocyte forms near the centromere and subsequent chiasmata form at a distance dependent on an interference effect exhibited by the first. An exhaustive study of Locusta chromosomes caused

Fox (1973) to elaborate this model such that a 'chiasma determination mechanism' passed along the chromosome at a constant rate starting from the telomeres and moving to the centromere. Interference is explained by the mechanism requiring a fixed amount of time to become 'recharged' after chiasma determination. Certainly this model explains the genetic map of the human X chromosome but Jones

145 pointed out that the spaced out distribution of chiasma does not exclude non-sequential models (Jones, 1974). In addition, he described a mutant in rye in v/hich chiasma distribution was random and interference abolished. This

implies that both chiasma localization and interference are under a single genetic control. h similar mutant in

Drosophila, mei-3 52, has also been described (Baker and

Carpenter, 1972). Jones concluded that interference and chiasma localization were a consequence of control of recombination rather than control mechanisms in their own right.

Electron microscopic analysis and three-dimensional reconstruction of meiotic cells has revealed structures which are strong candidates for the 'machinery' of recombination. Termed recombination nodules they were first described in Drosophila oocytes (Carpenter, 1975) and have subsequently been found in other species including human spermatocytes (Rasmussen and Holm, 1973).

Two types of nodule are seen in Drosophila♦ The first type are roughly spherical, appear late in pachytene and in number per nucleus, number per chromosome arm and distribution along each arm correspond './ell to reciprocal exchange events. The second type appear more ellipsoidal in shape and are seen earlier in pachytene. From their uniform distribution it is postulated that the sites of these structures correspond to gene conversion events. From examining the morphology and; distribution of nodules in meiotic mutants Carpenter has proposed that all the

146 properties for chiasma determination and formation lie in the nodule (Carpenter, 1079). Her hypothesis is that the earlier ellipsoidal nodules determine all recombinational events and the subsequent determination of a site for reciprocal exchange results in conversion of an ellipsoidal nodule to a spherical nodule. This structure then performs the enzymatic functions of strand isomerization and resolution. The mutants moi-210 and mei- 41 exhibit reduced exchange and have morphologically abnormal nodules which are reduced in numbers. Carpenter concludes that these mutants synthesize reduced numbers of functional nodules and those that form functionally are abnormal in their ability to respond to (or generate) the constraints regulating chromosomal locations of reciprocal events. In contrast, the mutant mei-9 exhibits hinhly reduced exchanges yet has morphologically normal nodules and a normal number of nodules. Carpenter postulates that in this mutant the nodules are deficient in some enzymatic process of recombination such as strand isomerization in the iieselson-Padding model (Peselson and Padding, 1975). The role that chromosome architecture might '''lay in determining chromosomal regions sensitive to recombination was stressed by Hulten in her demonstration of localized chiasmata in human spermatocytes (llulten, 1974). It is interesting in this respect to note that a region of the X chromosome that is apparently ’prone to meiotic recombination exists between the loci detected by 52A and

147 DX13 (Xq27 - Xqter). The same region also exhibits a physical lability in the manifestation of a fragile site

(brookwell and Turner, 1933).

From results demonstrating differing sensitivity of crossing over to translocation heterozygosity in different chromosomal regions, Roberts (1972) concluded that the initiation of synapsis was crucial to chiasma determination. The hypothesis that the short stretches of synaptonerna 1 complex formed early in leptotene were initiated by special repeated sequences was put forward by von bettstein (1971). He saw the construction of the synaptonema1 complex as a result of folding of these sequences onto each other to bring the element to a continuous thread. The distribution of such sequences along the chromosome might determine which parts of the chromosome are able to recombine more frequently. Maguire calculated the frequencies of recombination she would expect between chromosomes with limited genetic homology.

From higher than expected frequencies of recombinants obtained from crosses in maize between chromosomes of differing extents of homology in genetically marked regions, she proposed that chiasna were determined before synapsis was complete (Maguire, 1977). This indicates that regions labile to recombination are sites at which synapsis is initiated. Rasmussen and Holm (1930) speculated that the distribution of recinrocral exchange events is governed not just by the sites at which synapsis is initiated but also the rate at which synapsis occurs.

148 From the features of the recombination nodules and electron microscopic observations on synaptonemal complex formation they postulate a model delineating four constraints on chiasma determination;

1) A fairly constant total number of nodules are synthesized or assembled during zygotene.

2) A variation in the rate of chromosome pairing and synaptonemal complex formation. 3) A temporal variation in nodule availability during zygotene and pachytene.

4) A mechanism whereby nodules can be selectively removed from the synaptonemal complex during the zygotene- pachytene transition. In this way the sites at which reciprocal exchange events occur is governed both by the DMA and/or chromosome structure and by the recombination nodule. It is manifest that chiasma localization occurs across a wide range of species and is presumably an ancient device in evolution. In addition, changes in chiasma distributions and frequencies can be selected for (Rees and Dale, 1974). These observations have led Carson (1975) to the hypothesis that recombination cannot reach into every corner of the genome and he postulates two types of heritable genetic variability systems, an 'open' variability system and a 'closed' variability system. he proposes that the genetic components of the open variability system operate singly, relatively late in

149 development and are freely made available to the vagaries of natural selection or random drift by chance recombination. They are consequently the basis of easily adaptive differences found within a species. As a contrast, the closed variability system consists of blocks of genes which have come to be inseparably associated in a coadapted chromosome segment. Such a coadaptive arrangement has been termed 'internal balance' (father,

1943) or 'supergenes' (Darlington and Mather, 1949). .Although it is not a necessary consequence, such associations create a genetic climate for the development of mechanisms leading to chiasma localization. Carson sees the closed variability system as evolving early in the history of species and becoming essentially fixed in the form of normally unchangeable heteroses and internally balanced sets of genes which oblige the species to solve its problems in a certain way. As a consequence he proposes a non-gradualistic mode of speciation which can occur within a few generations by means of a population flush-crash-founder cycle. The flush phase is characterized by a release from natural selection which leads to the break up of coadaptations and supergenes of the closed variability system (such population flushes in nature are accompanied often by a release of bizarre genetic variability). A sudden change in the environment leads to a population crash with occasionally the survival of but a single propagule which may well be by chance alone as selection would not have enough time to act. This

150 founder is seen as a highly unusual recombinant coming out of the closed variabilty system which only survives by the absence of competition. The organization of a new closed variability system ensues when the expanding population is once again brought under natural selection.

A direct test of Carson's hypothesis has been made available in the grasshopper genus Caledia which exhibits differences in chiasma distribution both between species and between chromosomal taxa (Shaw and Knowles, 1976). Two chromosomal taxa of Caledia cantiva differ by seven pericentric rearrangements, heterochromatin and allozyme differences. The Moreton taxa has interstitial chiasmata whilst the Torresian taxa exhibits a strictly proxima1/distal chiasma pattern. This consequently implies differences in their closed variability systems according to Carson. Coates and Shaw (1992) examined the chiasma distribution in the generation of a cross between the two taxa. They found a repositioning of chiasmata in all chromosomes leading to maximum recombination in interstitial regions thus disrupting the presumed internal balance of coadapted gene combinations and leading to novel genotypes of unpredictable fitness. In an accompanying paper, Shaw et al. (1932) analysed directly the effects of this recombinational repatterning by estimating the relative viabilities of recombinant and non-recombinant chromosomes (identified by neterochromatic markers) through their distribution in offspring from

151 backcrosses of the F^ with both parent taxa and in the F . They found that although the F^ exhibited normal viability

and fertility, the F^ were completely inviable and the

backcross progeny exhibited a highly reduced viability. In addition, the relative viabilities of chromosomes in

backcross progeny corresponded well to the structure of the hybrid zone between the Moreton and Torresian taxa. They concluded that each chromosome of each taxa is internally coadapted in a functional sense and such

coadaptation is required for harmonious gene interactions within the same chromosomes during development. This induced their speculation that the recombination system

plays an integral and directing influence in maintaining karyotype uniformity. The selective factor which is responsible for the survival or extinction of variants

relates primarily not to extraneous environmental factors, but to the acceptability of the variant by the maternal genome which discriminately activates only those zygotic genomes which correspond to its own pattern of chromosomal organization. Rees and Dale (1074) considered the effect of recombinational repatterning in selected lines of

Festuca and bo1iurn populations in less drastic terms. They acknowledged the selective disadvantage of repatterned chromosomes and saw this as an overall dissipation and depletion of the heritable variation both in the potential and the free state. The observation of localized recombination in the human X chromosome provides circumstantial evidence for

152 the existence of coadapted genetic components locked in epistases. The inference from other species is clearly, however, that this pattern of recombination may have been preserved through the evolution of the species to maintain the internal balance of genetic components v/ithin the chromosome and also as a species barrier. One might infer that the v/ithin species variation in recombination pattern in Galedia (Shaw and Knowles, 1976) might also exist in homo Sapiens and this would explain the differences in recombination fraction observed between the same loci in different populations (Weitkamp, 1974).

153 CHAPTER 5 Summary and 'Perspectives

154 This research project has investigated the pattern of genetic recombination in the human X chromosome. It provides convincing evidence for Janssens' chiasnatype theory when comparisons are made to the chiasma distribution along male autosomes. In addition, it suggests non-uniformity of recombination frequency along the X chromosome. This may be an accidental result of chromosome structure or a consequence of the existence of regions inaccessible to crossing over. In this respect it would be interesting to examine the distribution of coding sequences along the 1C chromosome. A random distribution would support the hypothesis that genetic components may not recombine freely in all parts of the genome.

The construction of a genetic map of the ''/hole human

X chromosome permits linkage analysis between any of the markers used and any X-linked genotype. It has been estimated that the majority of loci must lie within 20cX of the marker loci analysed in this study. The delineation of aberrant X-linked genotypes to a small region of the chromosome permits diagnosis of such X-linhed disorders through the determination of the genetic constitution of the surrounding chromosome. In addition, analysis of RITA transcripts from this region in normal and affected individuals may allow the isolation of the particular D’TA an d Rd A segments responsible for the affected condition. This would then facilitate the indroduction of a functional allele into a mutant background once the technology for such 'gene therapy' exists.

155 CHAPTER 5 References

15 6 Ada, G . L . , Humphrey, J.H., Askonas, B . A. , HcDevitt, II. Q. and Hossal, G.J.V. (19G6) Exp. Cell Res. £1, 557-572

Bak, A. Leth, Zeuthen, J. and Crick, F.II.C. (1977) Proc. Uatl. Acad. Sci. USA 74, 1595-1599

Baker, B.S. and Carpenter, A.T.C. (1972) Genetics 71_, 255- 28 G

Baker, B.S., Carpenter, A.T.C., Esposito, 14.3. , Esposito, R.C. and Sandler, L . (1975) Ann. Rev. Genet. 10, 53-134 Benton, U.D. and Davis, R.U. (1977) Science 195,180-182

Benyajati, C. and T'orcel, A. (1976) Cell 9, 393-407

Blattner, F.R., Williams, B.G., Blechl, A.E., Denniston- Thompson, K., Fiiber, II. E. , Furlong, L.-A., Grunv/ald, D.J., Kiefer, D.O., Moore, D.D., Sheldon, H.L. and Smithies, O. (1977) Science 196, 161-169

Botstein, D., White, R . 7j . , Scolnick, M. and Davis, R.W. (1980) Arn. J. Hum. Genet. _32, 314-3 31

Boyer, H. and Roulland-Dussoix, D. (1969) J. Mol. Biol. 41, 459-472

Britten, R.J. and Kohne, D.E. (1968) Science 151, 529-540 Britten, R.J., Graham, D.E. and Ueufeld, R.R. (1974) Methods in Enzvmology 29E, 363-^06 Brookv/ell, R. and Turner, G. (1993) Hum. Genet. 63, 77 Brown, S. and Zonary, D. (1955) Genetics 40, 850-873

D r u f l a g , D . L. (1980) Ann. Rev. Genet. 14, 121-144

C a l l a n , H .G . and Perrv, P.E. (1977) Phil. Trans B o n d . B 277, 277-233

Camerino, G., Mattei, H . G . , Uattei, J . F . , Java, I'. and I'and el, J.-L. ( 1923) Mature 306, 701-704 Carpenter, A.T.C. (1975) Proc. Uatl. Acad. Sci. USA 72, 3186-3189 Carpenter, A.T.C. (1979) Chromosoma 7H5, 259-292 Carrano, A.V., Gray, J.U., Langlois, B.G., Burkhart- Schultz, K.J. and Van Dilla, U.A. (1979) proc. Uatl. Acad. Sci. 76, 1382-1384 Carson, U.L. (1975) Am. Uatur. 109, 93-92

157 Casadaban, M.J. and Cohen, S.M. (1930) J. Mol. Biol. 133, 179-201

Coates, D.J. and Shaw, D.D. (1932) Chromosoma 3^5, 509-531

Comings, D.H. (1972) Adv. Hum. Genet. 3, 237-243

Comings, D . F.. (1973) Ann. Rev. Genet. 12, 25-46 Creighton, H .B. and HcClintock, E. (1931) Proc. Hatl. Acad. Sci. USA 17, 492-497 Darling, S.M., Crarapton, J . H. and Uilliamson, R. (1932) J. Mol. Biol. 154, 51-53 Darlington, C.9. (1931) Biol. Rev. 6, 221-234 Darlington, C.D. and feather, K. (1949) The Elements of Genetics. /Alien and Unwin, London

Davidson, E.1I. and Britten, R.J. ( 197 3 ) 9. Rev. Biol. 43, 565-513 Davies, K.E. (1931) Hum. Genet. 53, 351-357 Davies, K.R., Young, R.D., Elies, R.G., Hill, H . H. and ’•Jill iamson, R. (1931) Mature 293, 374-375 Davies, K.E., Pearson, P.L., Harper, P.S., i'urrav, J.f ., O'Brien, T., Sarfarazi, ! ■. and Williamson, R. ( 1933 ) Duel. Acids Res. 11, 2303-2312

Davies, K.E., Hartley, p .A ., Hurray, J.H., Harper, P.S., Casey, G., Taylor, P. and Williamson R.(1933R) Cold Spring Harbor Banbury Reports Denhardt, D.T. (1966) Biochem. Biophvs. Res. Cominun. 23, 641-545 ~ " Doolittle, b.F. and Sapienza, C. (1930) Mature 234, 601- GO 3 Drayna, D., Davies, H . , 'hartley, D., Handel, J.-L., Carnerino, G., hi 11 iamson, R. and Tihite, R. (1984) Proc. Hatl. .Acad. Sci. USA, manuscript submitted

DuPraw, E.J. (1963) Cell and Molecular Biology. Academic Press, Dew York DuPraw, E.J. (1970) DMA and Chromosomes. Holt, Rinehart and Winston, Dew York Ferguson-Smith, M.A., Sanger, R., Tippett, P., Aitken, D . A . , and Boyd, E. ( 1932) Cytogenet. Cell Genet. 3_2, 273- 27 4

153 Finch, J.T. and Nlug, A. (1976) Proc. Natl. Acad. Sci. USA 73, 1397-1901 Fox, D.P. (1973) Chromosoma 43, 289-323

Gall, J.G. (1963) Nature 198, 36-38

Gall, J.G. and Pardue, M.L. (1969) Proc. Natl. /Acad. Sci. USA S3, 373-383 Gall, J.G. and Pardue, M.L. (1971) Methods in Enzymology 21, 470-480 Geever, P.F., I/ilson, L.B., Nallaseth, F.S., Milner, P.F., Bittner, M. and Wilson, J.T. (1981) Proc. Natl. Acad. Sci. USA 70, 5031-5035

Good fellow, P., Banting, G., Levy, R., Povev, S. and Mcl-iichael, A. (1930) Somat. Cell Genet. _6, 777-733 Good fellow, P., Banting, G. Trowsdale, J., Chambers, S. and Solomon, E. ( 1932) Proc. Natl. Acad. Sci. IJS/A 79, 1190-1194 Gosden, J.R., Mitchell, A.R., Buckland, R.A., Clayton, R.P. and Evans, li.J. ( 1975 ) Exp. Cell Res. 9_2, 143-158 Goss, S.J. and Harris, II. ( 1975) Nature 255, 680-684 Gusella, J.F., Keys, C., Varsanyi-Breiner, A., Kao, F-T., Jones, C., Puck, T.T. and iiousman, D. (1930) Proc. Natl. Sci. USA 77, 2829-2833 Gusella, J.F., Uexler, N.S., Conneally, P . r •., Naylor, S.L., Anderson, II. A., Tanzi, R . E. , Watkins, P.C., Ottina, K., Wallace, M.R., Sakaguchi, h. Y , , Young, A.B., Shoulson. I., Bonilla, E. and Martin, J.B. (1933) Nature 306, 234- 2 38

Haldane, J.B.S. (1931) Cytologia _3, 54-65

Hand, R. (1978) Cell 1_5, 317-325 Harper, M.E. and Saunders, G.F. (1981) Chromosona 8J3, 431- 439 Harper, IN E . , Barrera-Saldana, II. A. and Saunders, G.F. (1982) /Am. J. Hum. Genet. _3_1, 227-234 Ueintz, N., Zernik, M. and Boeder, R.G. (1981) Cell 24, 661-668 Holm, P.B. and Rasmussen, S.W. (1977) Carlsberg. Res. Commun . 4 2 , 2 8 3-323

159 Holmquist, G., Gray, [ . , Parker, T. and Jordan, J. (1982) Cel1 21' 121-129 Houck, C.M., Rinehart, F.P. and Schmid, C.b. (1979) J. ko1. Biol. 132, 289-306

Hozier, J.C., Renz, M. and Mehls, P. ( 1977) Chromosome 62, 301-318

Hulten, M . (1974) Hereditas jREp 55-73 Hulten, r.. , Palmer, R.U. and Laurie, D . A.. (1982) Ann. dura. Genet. 4_6, 167-175 ISCU (1981) .An International System for Human Cytogenetic 1'Ionenclature - High Resolution Banding. Cytogenet. Cell Genet. 3_1, 1-23 Igo-Remenes, T. and Zachau, II.G. (1977) Cold Spring Harbor Symp. Quant. Biol. 42:, 109-118 Igo-Kerrienes, T . , Slorz, V. and Zachau, H.G. ( 1982) Ann. Rev. Biochem. ^5^, 89-121 Jackson, D.A., Symons, R.H. and Berg, P. (1972) Proc. Hatl. Acad. Sci. USA 69, 2904-2909

Janssens, F.A. (1909) Cellule 2_5, 387-411

Jaye, 11. de la Salle, H., Schanber, F., Balland, A., Hohli, V., Fideli, A., Tolstoshev, P. and Lecocg, J.-P. (1983) Mucl. Acids Res. 11, 2325-2335 Jelinek, W.R., Toomey, T.P., Leinv/and, L., Duncan, (3. II., Biro, P.A., Choudary, P.V., Veissman, 5.4., Rubin, C.H., Houck, C.H., Deininger, P.L. and Schmid, C.b. (1980) Proc. Hatl. Acad. Sci. USA 11_, 1393-1402

Jelinek, I /.R. and Schmid, C.b. ( 1982) Ann. Rev. Biochem. 51, 813-844 Jepperson, P.G.LT. and Lankier, A.T. (1979) Fuel. Acids Res. 1_, '19-67 Jolly, D.J., Fsty, A.C., Bernard, U.U. and Friedmann, T. (1982) Proc. Hatl. Acad. Sci. USA 7_9, 5038-5041 Jones, G.H. (1974) nature 250, 147-143 Jones, K.b. (1973) In Hew Techniques in Biophysics and Cell Biology, Pain, R.H. and Smith, B.J., eds., pp29-56, J. Wiley and Sons, London Ran, Y.W. and Dozy, A.H. (1978) Lancet ii, 910-911

160 Kavenoff, R. and Zimin, B.H. ( 197 3) Chromosoma 4U, 1-20 Kingston, II. M., Thomas, W.S.T., Pearson, P.L., Sarfarazi, M. and Harper, P.S. (1933) J. Med. Genet. 20, 255-253

K u r n i t , D.M. and lloehn, H. (1979) A n n . Rev. Genet. 13, 2 3 5 - 2 5 3

L a e m m l i , U .K ., C h e n g , S .M ., Zidolnh, K . U., P a u l s o n , J .R ., Brown, J.A. and Baumbach, L’.R. (1977) Cold Spring Harbor Symp. Quant. Biol. 4_2, 351-355 Langer-Safer, P.R., Levine, M. and bard, D.C. (1982) Proc. Hat1. Acad. Sci. USA 79, 4331-4385

Latt, S.A. (1931) .Ann. Rev. Genet. _1_5, 11-55 Laurie, D .A., Palmer, R.U. and Hulten, H .A. (1932) Ann. Hun. Genet. 4j3, 233-244 Lebo, R.V., Chakravarti, A., Buetow, K.H., Cheung, M.-C., Gann, h., Cordell, B. and Goodman, II. (1983) Proc. Uatl. Acad. Sci. USA 80, 4308-4312

Lefevre, G., Jr. (1971) Genetics _67, 497-513 Littl e, P . F . R.. , Addison, G., barling, S., Uilliamson, R., Camba, L. and Lodell, B. (1980) Mature 235, 144-147 Lyon, I:. F. ( 1976) Genet. Res. _28, 291-300

IIcKusick, V . A. (19:30) J. Heredity 7_1, 370-391

McKusick , V.A. (1933) Uendelian Inheritance in Kan : Catalogs of /autosomal Dominant, Autosomal Recessive and K - linked Phenotypes, 6th edn.. Johns Hopkins University Press, Baltimore Maguire, K.P. (1977) Phil. Trans. R. Soc. Lond. B 277, 245-258 Malcolm, S., Barton, P., Murphy, C., Ferguson-Smith, K.A., Bentley, D.L. and Babbitts, T.H. (1982) Proc. Uatl. Acad. Sci. USA 19_, 4-957-4961 Maniatis, T., Hardison, R.C., Lacy, E., Lauer, J., O'Connell, C., Quon, D., Sim, G.K. and Ufstradiatis, A. (1978) Cell 15, 637-701

Maniatis, T ., Frit s ch , H .F . and S a m b r o o k , J . (1932) Mole c u l a r cloning (A laboratory nianua 1 ) . Cold Spring Harbor Laboratory Press, Hew York Manuel idis, L. and 'u, J.C.( 1973) Mature 276, 92-94

161 Manuelidis, L . and Biro, P.A. (1982) Nucl. Acids Res. 10, 3221-3239 Barsden, II. and Laemmli, U.K . (1979) Cell

Mather, X . (1933) Biol. Rev. 1_3, 252-292

lather, X . (1943) Biol. Rev. 1-8, 3 2-64 Maynard-Smith, J. (1978) The Evolution of Sex, Cambridge University Press I lend elsohn, H.L., Mayall, B.H., Bogart, H., tioore, D.U., r II and Perry, B .H. (1973) Science 179, 1126-112':)

Meselson, M . S . and Radding, C.M. (197 5) Proc. !: a 11. Acad. Sci. USA 72, 35:3-361

Miller, 0 . I. . , Jr. and Llamkalo, r . a (1972) Int. Rev. Cytol. 33 , 1-23

M o o r h e a d , P . S . , Howell, P.C., Heilman, . J . , Bat imps , D . H . and Hunge rford, D.A. (1960) Exn. Cell. R e s . 20, 613-616 Morgan, T.II. (1911) J. Exp. Zool. 11, 305-414 Morgan, T.H. and Cattell, E. (1912) J. Exn. Zool. _13, 79- 101

Morton, M . E. ( 19 56) <\n o. Hum. Genet. 1_, 277-231

Moses, M .J . ( 195 8 ) J Biophysic. Biochem. Cytol. 4, 6 3 3- 633

Muller, il.J. (1916) Am Uatur. 50, 193-221

Murray, J.M., Davies, K.E., Harper, P.S., Meredith, L,. , Mueller, C.R. and hi 11 ifimson, R. (1982) Mature 300, 69-71 Hurray, J.M. (1984) PhD thesis, University of Cardiff, Males Mabholz, U., Higgiano, V. and Bodmer, MM (1969) Mature 223, 358-363 Ueuffer, M.G. and Coe, E.U., Jr. (1976) In Handbook of Diochemical and Molecular Biology,3rd edn., G.D. Pasoan, Ed. Cleveland CRC Press

Orgel, L.E. and Crick, F.H.C. (1930) Mature, 234, 604-607 Ott, J. (19 74) Am. J . Hum. Genet. _26, 588-597 Oudet, P., Gross-Bellard, M. and Chambon, P. (1975) Cell

162 4, 201-300 Page, D., deMart invi 1 le, B. , Barker, D., Hyman, A., White, R.L., Francke, U. and Botstein, D. (1902) Proc. ITatl. Acad. Sci. USA 79, 5352-5356 Paulson, J.R. and Laemmli, U.K. (1977) Cell _12, 317-029

Pembrey, M.E., Davies, K.E., b'inter, R.M., Elies, R.G., Williamson, R., Fazzoni, T.A. and Calker, C. (1933) Arch. Dis. Child, in the press

Polani, P.E. (1972) Chromosoma 3_6, 343-374 Race, R.R. and Sanger, R. (1975) Blood Groups in Man, 6th edn. . Blackv/ell .Scientific Publications, Oxford Rasmussen, S.w. and Holm, P.B. (1973) Carlsberg. Res. Commun. 4_3, 27 5-3 27 Rasmussen, S.l . and Holm P . i3. (1930) Ilereditas 93, 137-215

Rees, H. and Dale, P.J. (1974) Chromosoma 47_, 335-351

Ricciuti, F.C. and Ruddle, F.II. ( 1973 ) Genetics 74, 661- 573 Rich, A. (1932) Cold Soring Harbor Symp. Quant. Biol. 47, 1-12 Rigby, P.J.W., Dieckrnann, M., Rhodes, C. and Berg, P. (1977) J. Mol. Biol. 113, 237-251 Ringertz, H.R. and Savage, R.E. (1975) Cell Hybrids. Academic Press, Iiew York Roberts, P .A. (1972) Genetics 71, 401-415

Ruddle, F.H. (1931) Mature 294, 115-120

Schalet, .A. and Lefevre, G., Jr. (1976) In ' kshburner and E. Hovitski (Eds.), The Genetics and iology of Drosophila. Academic Press, London, ppS43-902

Schmid, C.W. and Deininger, P.L. ( 197 5 ) Cell o_, 345-352 Seabright, I:. (1971) Lancet ii, 971-972 S h a f i t - 3 a g a r d o, Maio, J.J. and Drown, F.L. (1932) duel. Acids Res. IT), 3175-3193 Uhaw, D.D. (1974) Chromosoma 4^5, 36 5-374

Shaw, D.D. and Knovdes, G.R. (1975) Chromosome 59, 103-127

163 Shaw, D.D., Uilkinson, P. and Coates, D.J. (1982) Chromosoma 3G, 533-549

Solomon, E., Bobrov;, 15., Good fellow. P.N., Bodmer, MT.E., Swallow, D.M., Povey, S. and doe 1, I'.. (1975) Sornat. Cell Genet. 43, 241-248

Southern, E.M. (1975) J. Mol. Biol. 98, 503-517

Stadler, J., Larson, A., Engel, J.D., Dolan, I'., Groudine, M. and Ueintraub, II. (1900) Cell 2!0, 451-450 Steinmetz, H., Hinard,R., Horvath, S., EcHicholas, J., Srelinger, J., Uahe, C., Long, H., Mach, B. and Hood, L. (1982) Mature 300, 35-42 Stern, C. (1931) Biol. Sbl. _51, 547-587

Sturtevant, A.II. (1913) J. Exp. Zool. _14, 43-59

Sutton, \:.S. (1903) Biol. Bull. (Moods Hole) 4, 231-243 Szabo, P., Elder, R., Steffenson, D.M. and Uhlenbeck, O.C. (1977) J. Mol. Biol. 115, 539-553 Szabo, P. and Hard, D.C. (1982) Trends Diochem. Sci. 7, 425-427 “

Tariverdian,G. and Meek, B . (1982) Hum. Genet. G2, 95-109

Taylor, J.H. (1958) Sci. Am. 198, 36-47

Taylor, J.H. (1963 ) In J.II. Taylor (Ed.) Molecular Genetics. Academic Press, Hew York, pp65-113

Taylor, J.H. (1965) J. Cell. Biol. 25, 57-57 Tease, C. and Jones, G.H. (1978) Chromosoma 59, 163-178 Tiemeier, D ., Enquist, E. and Leder, P. (1976) Mature 263, 526-527

Trent, J.M., Olson, S. and Lawn, R.H. ( 1982) Proc. Mratl. Acad. Sci. USA 22/ 7309-7013 vorT 'ettstein, D. (1971) Proc. Natl. Acad. Sci. MTS A 53, 351-855 Mahl, G.H. , Stern, M:. and Stark, G.R. ( 1979) Proc. Hatl. Acad. Sci. (JSA 7_6, 3683-3637 Heatherall, D.J. and Clegg, J.3. (1982) Cell 29, 7-9 Heismann, A. (1887) Mature Lond. 36, 607-609

154 Weiss, IRC. and Green, II. (1967) Proc. llatl. Acad. Sci. USA 59, 1104-1111

Weitkamp, L.R. (1974) Cytogenet. Cell Genet. 1^3, 179-132 I Jes tergaard, II. and von Wettstein, D. ( 1972 ) Ann. Rev. Genet. j5, 71-110 Wetrnur, J.G. ( 1975 ) Biopolymers L4, 2517-2524

White, K.J.D. (1973) /Animal cytology and evolution, 3rd edn.. Cambridge University °ress

1. ieacker,P ., Davies, K.E., Cooke, II.J., Pearson, P.L., ’./illiamson, R. , Southern, E., Zimmer, J., and Ropers,II.-II. (1933) Am. J. Hum. Genet., in the press Willard, H.F., Smith, K.D. and Sutherland, J. (1982) duel. Acids Res. 11, 2017-2033 Winder, P.E. and Steffensen, D.U (1973) Ann. Rev. Genet. 1_, 205-223 hood, W.B. ( 1936) J. Hoi. Riol. IjS, 113-133

W;u, J.C. and Ilanuelidis, L. (1930) J. Uol. Biol. 112, 3 5 3- 3 36 Yang, T.P., Hansen, S.R., Oishi, R.W., Ryder, D.A. and Hamkalo, B.A. (1982) Proc. Uatl. Acad. Sci. USA 7_9, 6593- 5597

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