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The Promise of Human Genome Editing for Rare and Genetic Disease Summary Report of the 2019 FORUM Annual Lecture
The promise of human genome editing for rare and genetic disease Summary report of the 2019 FORUM Annual Lecture The Academy of Medical Sciences The Academy of Medical Sciences is the independent body in the UK representing the diversity of medical science. Our mission is to promote medical science and its translation into benefits for society. The Academy’s elected Fellows are the United Kingdom’s leading medical scientists from hospitals, academia, industry and the public service. We work with them to promote excellence, influence policy to improve health and wealth, nurture the next generation of medical researchers, link academia, industry and the NHS, seize international opportunities and encourage dialogue about the medical sciences. Opinions expressed in this report do not necessarily represent the views of all participants at the event, the Academy of Medical Sciences, or its Fellows. All web references were accessed in February 2020. This work is © Academy of Medical Sciences and is licensed under Creative Commons Attribution 4.0 International. The Academy of Medical Sciences 3 The promise of human genome editing for rare and genetic disease Summary report of the 2019 FORUM Annual Lecture Contents Executive summary .................................................................................................... 4 An introduction to genome editing ................................................................................ 6 Genome editing: moving to the clinic ........................................................................... -
SRM Phd Dissertation FINAL
UCSF UC San Francisco Electronic Theses and Dissertations Title The mechanistic role of the clathrin light chain subunits in cell function Permalink https://escholarship.org/uc/item/9cj4b9dx Author Majeed, Sophia Rafaa Publication Date 2015 Peer reviewed|Thesis/dissertation eScholarship.org Powered by the California Digital Library University of California The mechanistic role of the clathrin light subunits in cell function by Sophia R. Majeed DISSERTATION Submitted in partial satisfaction of the requirements for the degree of DOCTOR OF PHILOSOPHY in Pharmaceutical Sciences and Pharmacogenomics in the GRADUATE DIVISION of the UNIVERSITY OF CALIFORNIA, SAN FRANCISCO Copyright 2015 By Sophia Rafaa Majeed ! ii! Acknowledgements ! I would like to thank my PhD thesis advisor, Dr. Frances Brodsky, for all of her support and scientific guidance throughout my time in graduate school. Frances has provided me with mentorship when I needed it, and has given me the freedom to think independently and pursue my own scientific ideas. She has constantly challenged me to think creatively and tackle big problems that don’t have simple answers. As a woman and as a scientist, Frances has inspired me to persevere in the face of challenges. She has taught me that even the most insurmountable difficulties can be overcome with the right combination of diligence, passion and determination. Thank you for providing me with the tools to become a critical and creative independent researcher. I would like to thank my other mentors who have had a profound impact on me, both on a scientific and personal level, during my time at UCSF. I would like to thank Drs. -
Since January 2020 Elsevier Has Created a COVID-19 Resource Centre with Free Information in English and Mandarin on the Novel Coronavirus COVID- 19
Since January 2020 Elsevier has created a COVID-19 resource centre with free information in English and Mandarin on the novel coronavirus COVID- 19. The COVID-19 resource centre is hosted on Elsevier Connect, the company's public news and information website. Elsevier hereby grants permission to make all its COVID-19-related research that is available on the COVID-19 resource centre - including this research content - immediately available in PubMed Central and other publicly funded repositories, such as the WHO COVID database with rights for unrestricted research re-use and analyses in any form or by any means with acknowledgement of the original source. These permissions are granted for free by Elsevier for as long as the COVID-19 resource centre remains active. Available online at www.sciencedirect.com ScienceDirect Editorial overview: Membrane traffic in the time of COVID-19 Frances M. Brodsky and Jennifer L. Stow Current Opinion in Cell Biology 2020, 65:iii–v This overview comes from a themed issue on Membrane Trafficking Edited by Frances M. Brodsky and Jennifer L. Stow https://doi.org/10.1016/j.ceb.2020.09.003 0955-0674/© 2020 Published by Elsevier Ltd. Frances M. Brodsky We write this editorial emerging from lockdown in countries across the Division of Biosciences, University College world in the face of the COVID-19 pandemic. These have been chal- London, Gower Street, London, WC1E 6BT, lenging, frightening, and too often catastrophic times for many. Such times UK lead to evaluation of one’s own enterprise in the context of a global *Corresponding author: Brodsky, Frances M. -
BBSRC Annual Report and Accounts 2007-08 HC
ANNUAL REPORT & ACCOUNTS 2007-2008 Biotechnology and Biological Sciences Research Council ANNUAL REPORT & ACCOUNTS 2007-2008 Presented to Parliament by the Secretary of State, and by the Comptroller and Auditor General, in pursuance of Schedule 1, Sections 2 [2] and 3 [3] of the Science and Technology Act 1965. Ordered to be printed by the House of Commons 16 July 2008. HC761 LONDON: The Stationery Office £18.55 Contents PART 1: MANAGEMENT COMMENTARY Chairman’s statement 1 Chief Executive’s report 3 Supporting world class research 6 Key funding data 7 Embedding Systems Biology 11 Major collaborative and multidisciplinary programmes 12 Delivering economic and social benefits 14 Collaborative research with industry 14 Commercialising research outputs 17 Tackling major challenges 20 People, skills, training and knowledge flows 22 Embedding our science in society 26 Opinion gathering and public dialogue 26 Outreach and engagement 28 Engaging young people in science 29 Corporate information 30 2007-2008 Council 30 Boards, Panels and Committees 31 Organisational developments 36 Financial review 39 Remuneration report 42 PART 2: ANNUAL ACCOUNTS Financial statements for the year ended 31 March 2008 45 This Annual Report covers the period from 1 April 2007 to 31 March 2008. BBSRC ANNUAL REPORT & ACCOUNTS BBSRC ANNUAL The Biotechnology and Biological Sciences Research Council (BBSRC), established by Royal Charter in 1994, is the UK’s principal funder of basic and strategic research across the biosciences (www. bbsrc.ac.uk). It is funded primarily by the Science Budget, through the Department for Innovation, Universities and Skills (DIUS). Our mission is to support high-class science and research training, and to promote knowledge transfer in support of bio-based industries and public engagement in bioscience. -
Winter for the Membership of the American Crystallographic Association, P.O
AMERICAN CRYSTALLOGRAPHIC ASSOCIATION NEWSLETTER Number 4 Winter 2004 ACA 2005 Transactions Symposium New Horizons in Structure Based Drug Discovery Table of Contents / President's Column Winter 2004 Table of Contents President's Column Presidentʼs Column ........................................................... 1-2 The fall ACA Council Guest Editoral: .................................................................2-3 meeting took place in early 2004 ACA Election Results ................................................ 4 November. At this time, News from Canada / Position Available .............................. 6 Council made a few deci- sions, based upon input ACA Committee Report / Web Watch ................................ 8 from the membership. First ACA 2004 Chicago .............................................9-29, 38-40 and foremost, many will Workshop Reports ...................................................... 9-12 be pleased to know that a Travel Award Winners / Commercial Exhibitors ...... 14-23 satisfactory venue for the McPherson Fankuchen Address ................................38-40 2006 summer meeting was News of Crystallographers ...........................................30-37 found. The meeting will be Awards: Janssen/Aminoff/Perutz ..............................30-33 held at the Sheraton Waikiki Obituaries: Blow/Alexander/McMurdie .................... 33-37 Hotel in Honolulu, July 22-27, 2005. Council is ACA Summer Schools / 2005 Etter Award ..................42-44 particularly appreciative of Database Update: -
Astex Pharmaceuticals Announces the Appointment of President Harren Jhoti, Phd to the Bioindustry Association Board of Directors
October 10, 2012 Astex Pharmaceuticals Announces the Appointment of President Harren Jhoti, PhD to the BioIndustry Association Board of Directors DUBLIN, Calif., Oct. 10, 2012 (GLOBE NEWSWIRE) -- Astex Pharmaceuticals, Inc. (Nasdaq:ASTX), a pharmaceutical company dedicated to the discovery and development of novel small molecule therapeutics, announced that Harren Jhoti, PhD, president and director of Astex Pharmaceuticals, was appointed to the BioIndustry Association (BIA) Board of Directors. About Harren Jhoti, PhD Dr. Harren Jhoti has served as Astex Pharmaceuticals president and member of the Board of Directors since the company's formation in July 2011. He co-founded Astex Therapeutics in 1999 and was chief scientific officer until November 2007 when he was appointed chief executive. Dr. Jhoti was awarded the Prous Institute-Overton and Meyer Award for New Technologies in Drug Discovery by the European Federation for Medicinal Chemistry in 2012 and was also named by the Royal Society of Chemistry as "Chemistry World Entrepreneur of the Year" for 2007. He has published widely including in leading journals such as Nature and Science and has also been featured in TIME magazine after being named by the World Economic Forum a Technology Pioneer in 2005. Dr. Jhoti served as a non-executive director of Iconix Inc. Before starting up Astex Therapeutics in 1999, he was head of Structural Biology and Bioinformatics at GlaxoWellcome in the United Kingdom (1991-1999). Prior to Glaxo, Dr. Jhoti was a post-doctoral scientist at Oxford University. He received a BSc (Hons) in Biochemistry in 1985 and a PhD in Protein Crystallography from the University of London in 1989. -
Anew Drug Design Strategy in the Liht of Molecular Hybridization Concept
www.ijcrt.org © 2020 IJCRT | Volume 8, Issue 12 December 2020 | ISSN: 2320-2882 “Drug Design strategy and chemical process maximization in the light of Molecular Hybridization Concept.” Subhasis Basu, Ph D Registration No: VB 1198 of 2018-2019. Department Of Chemistry, Visva-Bharati University A Draft Thesis is submitted for the partial fulfilment of PhD in Chemistry Thesis/Degree proceeding. DECLARATION I Certify that a. The Work contained in this thesis is original and has been done by me under the guidance of my supervisor. b. The work has not been submitted to any other Institute for any degree or diploma. c. I have followed the guidelines provided by the Institute in preparing the thesis. d. I have conformed to the norms and guidelines given in the Ethical Code of Conduct of the Institute. e. Whenever I have used materials (data, theoretical analysis, figures and text) from other sources, I have given due credit to them by citing them in the text of the thesis and giving their details in the references. Further, I have taken permission from the copyright owners of the sources, whenever necessary. IJCRT2012039 International Journal of Creative Research Thoughts (IJCRT) www.ijcrt.org 284 www.ijcrt.org © 2020 IJCRT | Volume 8, Issue 12 December 2020 | ISSN: 2320-2882 f. Whenever I have quoted written materials from other sources I have put them under quotation marks and given due credit to the sources by citing them and giving required details in the references. (Subhasis Basu) ACKNOWLEDGEMENT This preface is to extend an appreciation to all those individuals who with their generous co- operation guided us in every aspect to make this design and drawing successful. -
Lysosomes & Endocytosis
Lysosomes & Endocytosis Gordon Research Conference Molecular Mechanisms and Functions of Endosomal and Lysosomal Pathways June 12-17, 2016 Proctor Academy Andover, NH Chair: Roberto Weigert Vice Chair: Lois S. Weisman Contributors Meeting Program Sunday 2:00 pm - 9:00 pm Arrival and Check-in 6:00 pm Dinner 7:30 pm - 7:40 pm Welcome / Introductory Comments by GRC Site Staff 7:40 pm - 9:30 pm Endocytic Machinery at the Plasma Membrane Discussion Leaders: Sandra Schmid (University of Texas Southwestern Medical Center, USA) and Mark Von Zastrow (University of California, San Francisco, USA) 7:40 pm - 8:00 pm Volker Haucke (Leibniz-Institut für Molekulare Pharmakologie, Germany) "Phosphoinositide Conversion Within the Endolysosomal System" 8:00 pm - 8:10 pm Discussion 8:10 pm - 8:30 pm Ludger Johannes (Institut Curie, France) "Using Glycosphingolipids to Build Endocytic Pits in Clathrin-Independent Endocytosis" 8:30 pm - 8:40 pm Discussion 8:40 pm - 9:00 pm Margaret Robinson (University of Cambridge, United Kingdom) "Coated Vesicle Adaptors" 9:00 pm - 9:10 pm Discussion 9:10 pm - 9:25 pm Sara Sigismund (IFOM, The FIRC Institute for Molecular Oncology Foundation, Italy) "ER-PM Contact Sites Control EGFR Endocytosis" 9:25 pm - 9:30 pm Discussion Monday 7:30 am - 8:30 am Breakfast 9:00 am - 12:30 pm Endosomal Sorting and Recycling Discussion Leaders: Elizabeth Conibear (University of British Columbia, Canada) and Scott Emr (Cornell University, USA) 9:00 am - 9:20 am Peter Cullen (University of Bristol, United Kingdom) "Molecular Mechanisms of -
Binding and Immunoprecipitation (Phytohemagglutinin/HLA-DR Antigens/HLA-A, -B Antigens) JORDAN S
Proc. NatL Acad. Sci. USA Vol. 79, pp. 6641-6645, November 1982 Immunology Expression of Ia-like antigens by human vascular endothelial cells is inducible in vitro: Demonstration by monoclonal antibody binding and immunoprecipitation (phytohemagglutinin/HLA-DR antigens/HLA-A, -B antigens) JORDAN S. POBER AND MICHAEL A. GIMBRONE, JR. Laboratory ofVascular Pathophysiology, Departments of Pathology, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts 02115 Communicated by Baruj Benacerraf, July 6, 1982 ABSTRACT The expression of Ia-like antigens by cultured clonal antibodies has suggested that endothelial cells may lack human endothelial cells has been investigated by means ofmono- Ia-like antigens (16). clonal antibody binding to intact cells and by immunoprecipitation The expression of HLA-DR or other Ia-like antigens by vas- ofradioiodinated membrane proteins. Primary growing and con- cular endothelium would have at least two important biological fluent cultures of human umbilical vein endothelium express lit- implications. First, the presence of Ia-like antigen-bearing en- tle, ifany, detectable Ia-like antigens under standard culture con- dothelial cells within an organ graft could significantly enhance ditions. However, treatment of primary cultures with the lectin its immunogenicity and hence its likelihood of transplant re- phytohemagglutinin induces the expression of Ia-like antigens. jection (17). In the mixed reaction, a model oftrans- This action of the lectin uniformly affects all the endothelial cells lymphocyte in a culture, does notdepend on cell division, and is associated with plant rejection, Ia-like antigens on the stimulator lymphocyte a cell shape change. The data presented in this report provide population serve as the major provokers ofallogeneic responder unequivocal serological and biochemical demonstration of Ia-like lymphocyte proliferation (18). -
Human B-Cell Alloantigens DC1, MT1, and LB12 Are Identical to Each
Proc. NatL Acad. Sci. USA Vol. 78, No. 7, pp. 4566-4570, July 1981 Immunology Human B-cell alloantigens DC1, MT1, and LB12 are identical to each other but distinct from the HLA-DR antigen (major histocompatibility complex/two-dimensional gel electrophoresis/polymorphism) DEBORAH A. SHACKELFORD*, DEAN L. MANNt, JON J. VAN ROODf, G. B. FERRARA§, AND JACK L. STROMINGER* *The Biological Laboratories, Harvard University, Cambridge, Massachusetts 02138; tThe Immunology Branch, National Cancer Institute, National Institutes of Health, Bethesda, Maryland 20205; tDepartment of Immunohaematology, University Medical Center, Leiden, the Netherlands; and §Immunohematological Research Center, AVIS Bergamo and Ospedale Generale Provinciale, Massa, Italy Contributed by Jack L. Strominger, April 27, 1981 ABSTRACT Some human B-lymphoblastoid cell lines are Table 1. Serological reagents shown to express at least two types ofIa-like antigens. One antigen is defined by alloantisera to HLA-DR, and the other antigen is Laboratory HLA-DR Other defined by alloantisera and a monoclonal antibody to the speci- of specificity local ficities DC1, MT1 (MB1), and LB12, which are in linkage dis- Antiserum origin associations specificity equilibrium with HLA-DR. The subunits of the DC1 molecule Ia715 Mann DR1,2,w6 MT1(MBl) differ from those of the DR molecule. The light chains of both DR16213.3 molecules are structurally polymorphic. (anti-LB12) van Rood DR1,2,w6 LB12 Fel31/6 Ferrara (14) DR1,2,w6 DC1 The HLA-D/DR region of the human major histocompatibility Fe77/43 Ferrara (14) DR1,2,w6 DC1 complex (MHC) controls the expression of a noncovalent com- Genox 3.53 Bodmer (28) DR1,2,w6 plex oftwo membrane glycoproteins ofMr 34,000 and 29,000 JongBles 12505 (1). -
Binding and Immunoprecipitation (Phytohemagglutinin/HLA-DR Antigens/HLA-A, -B Antigens) JORDAN S
Proc. NatL Acad. Sci. USA Vol. 79, pp. 6641-6645, November 1982 Immunology Expression of Ia-like antigens by human vascular endothelial cells is inducible in vitro: Demonstration by monoclonal antibody binding and immunoprecipitation (phytohemagglutinin/HLA-DR antigens/HLA-A, -B antigens) JORDAN S. POBER AND MICHAEL A. GIMBRONE, JR. Laboratory ofVascular Pathophysiology, Departments of Pathology, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts 02115 Communicated by Baruj Benacerraf, July 6, 1982 ABSTRACT The expression of Ia-like antigens by cultured clonal antibodies has suggested that endothelial cells may lack human endothelial cells has been investigated by means ofmono- Ia-like antigens (16). clonal antibody binding to intact cells and by immunoprecipitation The expression of HLA-DR or other Ia-like antigens by vas- ofradioiodinated membrane proteins. Primary growing and con- cular endothelium would have at least two important biological fluent cultures of human umbilical vein endothelium express lit- implications. First, the presence of Ia-like antigen-bearing en- tle, ifany, detectable Ia-like antigens under standard culture con- dothelial cells within an organ graft could significantly enhance ditions. However, treatment of primary cultures with the lectin its immunogenicity and hence its likelihood of transplant re- phytohemagglutinin induces the expression of Ia-like antigens. jection (17). In the mixed reaction, a model oftrans- This action of the lectin uniformly affects all the endothelial cells lymphocyte in a culture, does notdepend on cell division, and is associated with plant rejection, Ia-like antigens on the stimulator lymphocyte a cell shape change. The data presented in this report provide population serve as the major provokers ofallogeneic responder unequivocal serological and biochemical demonstration of Ia-like lymphocyte proliferation (18). -
Differentiation Antigens, Hlaand .82-Microglobulin
Proc. NatL Acad. Sci. USA Vol. 80, pp. 524-528, January 1983 Immunology Stable transformation of mouse L cells for human membrane T-cell differentiation antigens, HLA and .82-microglobulin: Selection by fluorescence-activated cell sorting (gene cloning/Leu-1, Leu-2 transformants) PAULA KAVATHAS AND LEONARD A. HERZENBERG Department of Genetics, Stanford Univeristy School of Medicine, Stanford, California 94305 Contributed by Leonard A. Herzenberg, October 12, 1982 ABSTRACT We isolated stable transformants ofmouse L cells To enrichfor transformants in general, we cotransformed mouse expressing human cell surface differentiation antigens by using L cells with total cellular DNA and with the herpes simplex immunofluorescence with monoclonal antibodies and selection thymidine kinase (TK) gene and selected for TK' transformed with a fluorescence-activated cell sorter (FACS). Mouse L cells cellsbygrowthinhypoxanthine/aminopterin/thymidine (Hyp/ (TK-) were cotransformed with human cellular DNA and the Apt/Thd) medium. Initially, HLA class I and human p2-mi- herpes simplex virus thymidine kinase (TK) gene. TKV transfor- croglobulin (132m) transformants were selected to establish pro- mants were first selected. The TKV populations were stained with cedures for selecting rare transformants from populations of various fluorescent antibodies to membrane antigens, and positive TKV transformed cells. Later, selection for transformants ex- cells were sorted and cloned by using a FACS. Transformants for T-cell differentiation was The fre- HLA class I antigens, for f32-microglobulin, and for the T-cell dif- pressing antigens imposed. ferentiation antigens Leu-1 and Leu-2 were isolated. The fre- quency of antigen transformants for both Leu-1 and Leu-2 an- quency of antigen transformants among the TKV transformants tigens among the TKV transformants was about 0.5 x 10-3.