DOCSLIB.ORG

Gleevec's Glory Days

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text
Gleevec's Glory Days Diabetes Detectives || $$$ to Databases || Anthrax 101 || Fighting Parasites in Bangladesh DECEMBER 2001 Gleevec’s Glory Days The Long Journey of a Celebrated Anticancer Drug FEATURES Gleevec’s Glory Days Mirpur’s Children 10 22 An apparent overnight success, this new A slum in Bangladesh yields clues about leukemia drug has decades of research behind it. immunity, infection and an illness that afflicts By Jill Waalen millions worldwide. By David Jarmul 16 Confronting Diabetes From All Angles 26 Scientific Outliers To combat this growing epidemic, researchers are hunting for genes, exploring cell-signaling How teachers and students in rural America pathways and looking at obesityÕs role. can learn good science. By Karen Hopkin By Mitch Leslie 16 In Starr County, Texas, an alarming 2,500 Mexican- American residents have type 2 diabetes. These women, at the Starr County Health Studies Office, undergo regular monitoring of their disease, which has strong genetic and evironmental components. DEPARTMENTS 2 NOTA BENE 30 HANDS ON Howard Hughes Medical Institute Bulletin ulletin Building Interest in the December 2001 || Volume 14 Number 5 3 PRESIDENT’S LETTER Human Body HHMI TRUSTEES Biomedical Research in a James A. Baker, III, Esq. Changed World Senior Partner, Baker & Botts NEWS AND NOTES Alexander G. Bearn, M.D. Executive Officer, American Philosophical Society 32 To Think Like a Scientist Adjunct Professor, The Rockefeller University UP FRONT Professor Emeritus of Medicine, Cornell University Medical College Frank William Gay 4 Mouse Model Closely 33 Undergraduate Taps Into Former President and Chief Executive Officer, summa Corporation James H. Gilliam, Jr., Esq. Mimics Human Cancer Tomato Communication Former Executive Vice President and General Counsel, Beneficial Corporation 6 Database Science Forcing New Hanna H. Gray, Ph.D., CHAIRMAN 34 A Course Set by Example President Emeritus and Harry Pratt Judson Distinguished Service Standards for Openness Professor of History, The University of Chicago Garnett L. Keith 34 Who’s Who in Genes and Chairman, SeaBridge Investment Advisors, L.L.C. 9 Anthrax 101: A Conversation Former Vice Chairman and Chief Investment Officer, The Prudential Gender Research Insurance Company of America With William Dietrich Jeremy R. Knowles, D.Phil. Dean of the Faculty of Arts and Sciences and Amory Houghton 35 A Rising Star in Gene Therapy Professor of Chemistry and Biochemistry, Harvard University William R. Lummis, Esq. Former Chairman of the Board of Directors 36 Smarter Waste Management and Chief Executive Officer, The Howard Hughes Corporation Anne M. Tatlock Chairman and Chief Executive Officer 37 Lost in World Trade Fiduciary Trust Company International Center Attack HHMI OFFICERS Thomas R. Cech, Ph.D., President Peter J. Bruns, Ph.D., Vice President for Grants and Special Programs 38 HHMI LAB BOOK David A. Clayton, Ph.D., Vice President for Science Development Stephen M. Cohen, Vice President and Chief Financial Officer Joan S. Leonard, Esq., Vice President and General Counsel 40 CLOSE-UP Gerald M. Rubin, Ph.D., Vice President for Biomedical Research Nestor V. Santiago, Vice President and Chief Investment Officer Skin Bares Its Secrets HHMI BULLETIN STAFF Cori Vanchieri, Editor PERSPECTIVE Jim Keeley, Science Editor 42 Jennifer Donovan, Education Editor Diabetes in High-Risk Patricia Foster, Manager of Publishing Minorities Kimberly Blanchard, Editorial Coordinator 22 Elizabeth Cowley, Copy Editor Maya Pines, Contributing Editor 43 INSIDE HHMI 6 Steven Marcus, Peter Tarr, Laurie Vaughan, story editing To Pay for Biomedical Research, Kathy Savory, copy editing Buy Low, Sell High and Act Fast David Herbick Design, Publication Design Telephone (301) 215 8855 n Fax (301) 215 8863 n www.hhmi.org The Bulletin is published by the HHMI Office of Communications, 44 IN MEMORIAM Robert A. Potter, Director. A Tribute to Irving S. Shapiro © 2001 Howard Hughes Medical Institute Letters to the editor: We invite your comments. Send your letters via e-mail to [email protected] or regular mail to Letters, Office of Communications, Howard Hughes Medical Institute, 4000 Jones 45 HHMI ONLINE Bridge Road, Chevy Chase, MD 20815-6789. We reserve the right to edit for space and clarity. Please also include your name, address Order hhmi Materials Online (e-mail or postal) and phone number. The opinions, beliefs and viewpoints expressed by authors in the HHMI Bulletin do not necessarily reflect the opinions, beliefs and viewpoints or DAN COHEN (LEFT), FAKRUL ALAM (TOP RIGHT), STUART BRADFORD (BOTTOM RIGHT) (BOTTOM BRADFORD STUART RIGHT), ALAM (TOP FAKRUL COHEN (LEFT), DAN On the Cover: Illustration by Stuart Bradford official policies of the Howard Hughes Medical Institute. NOTA BENE onic stem cells to create strains of mice in Medicine of the National Academies. I Stephen P. Bell, an hhmi investigator at which a gene is disabled, or “knocked out.” the Massachusetts Institute of Technology, I Robert J. Lefkowitz, an hhmi investiga- won the 2001 American Society for Bio- I Ana Belén Elgoyhen and Hugo J.F. Maccioni, tor at Duke University Medical Center, chemistry and Molecular Biology/Schering- hhmi international research scholars in received the Jessie Stevenson Kovalenko Plough Research Institute Award for Argentina, won the 2000 Bernardo A. Hous- Medal from the National Academy of contributions to biochemistry and molecu- say Award from the Argentine Biological Sciences. The award is given approximately lar biology. Society. The award is given biennially to every three years for contributions to the recognize contributions to biological sci- medical sciences. Lefkowitz also received I Günter Blobel, an hhmi investigator at ences research. the Louis and Artur Lucian Award for The Rockefeller University, was named a Research in Circulatory Diseases from member of the Orden Pour le Mérite, a I Joseph Heitman, an hhmi investigator McGill University in Montreal. German honorary society. Membership is at Duke University Medical Center, won limited to 30 Germans and 30 foreign the 2002 American Society for Biochem- I Ben Margolis, an hhmi investigator at members who have been recognized inter- istry and Molecular Biology–Amgen the University of Michigan Medical School, nationally for their contributions to science Award. It is given in recognition of a new won the university’s 2001 Faculty Recogni- and culture. investigator’s achievements in applying tion Award. biochemistry and molecular biology to the I Patrick O. Brown, an hhmi investigator understanding of human disease. I Nebraska Wesleyan University won the at Stanford University School of Medicine, 2001 Council of Independent Colleges received the 2001 Millennium Pharma- I Barry Honig, an hhmi investigator at Heuer Award, funded by the Russell Pearce ceuticals Distinguished Achievement Award Columbia University College of Physicians and Elizabeth Crimian Heuer Foundation, for Genomics Research in Clinical and Surgeons, will receive the 2002 for a program supported by a grant Immunology. Founders Award from the Biophysical from hhmi. The award, recognizing Society, recognizing his achievements in achievement in undergraduate science edu- I R. David Bynum, director of the hhmi- biophysics. cation, is given to two institutions in the supported undergraduate biological United States. sciences education program at the State I Arthur L. Horwich, an hhmi investigator University of New York at Stony Brook, at Yale University School of Medicine, won I Daphne Preuss, an hhmi investigator at received the American Society for Cell the 2001 Hans Neurath Award from the the University of Chicago, won the 2001 Biology’s 2001 Bruce Alberts Award for Protein Society for his contributions to American Society for Cell Biology/Promega Excellence in Science Education. He is an basic research in protein science. Early Career Life Scientist Award. Another associate professor of biochemistry. hhmi investigator, Erin O’Shea,University I Four teachers from the Fred Hutchinson of California, San Francisco, won the award I Kevin P. Campbell, an hhmi investigator Cancer Research Center Science Education in 2000. at the University of Iowa College of Partnership, supported by a grant from Medicine, received the 2001 S. Mouchly hhmi, were honored for teaching excel- I Joseph S. Takahashi, an hhmi Small Scientific Achievement Award from lence. Krestin Bahr, MaryMargaret Welch investigator at Northwestern University, the Muscular Dystrophy Association. and David Ziegler won the 2001 Washing- received the W. Alden Spencer Award The award recognizes a researcher whose ton Award for Excellence in Education. Jim from Columbia University College of work in neuromuscular disorders shows Boyce won the 2001 Christa McAuliffe Fel- Physicians and Surgeons for contributions particular promise. lowship, named in honor of the first teacher to neural research. selected to travel in space. I Mario R. Capecchi, an hhmi investigator I Wayne M. Yokoyama, an hhmi investiga- at the University of Utah, won the 2001 I Investigators Thomas M. Jessell, Columbia tor at Washington University School of Albert Lasker Award for Basic Medical University College of Physicians and Sur- Medicine, shared the 2001 Novartis Prize Research. He shared the award with Martin geons; Douglas A. Melton, Harvard University; for Basic Immunology with Klas Kärre of Evans, Cardiff University, Wales, and Oliver Bert Vogelstein, The Johns Hopkins Universi- Sweden and Lorenzo Moretta of Italy. The Smithies, University of North Carolina at ty School
Load more

Recommended publications
  • Harnessing the Power of Precision Oncology
    Cancer Advances Cleveland Clinic Cancer Center | Winter 2019 Harnessing the Power of Precision Oncology Ranked No. 5 in America for cancer care by U.S. News & World Report. Dear colleagues, Welcome to this issue of Cancer Advances. Our cover story features a sampling of our work in genetics and Advancing genomics, which is shifting the focus of questioning in oncologic research and care from tumor location to genetic mutation. Our researchers are approaching questions of cancer genetics from across the continuum, including detecting cancers at an earlier stage (p. 4), best Precision practices in testing for non-small cell lung cancer (p. 6), expanding the use of predictive assays (p. 7), treatments targeting individual tumor DNA (p. 8) and a new National Cancer Institute grant to study response prediction in Oncology radiation oncology (p. 10). Our leadership in developing the accreditation program for rectal cancer (p. 22) and the continued relevance and from Risk Prediction utility of the Khorana score (p. 14) showcase our ability to determine the line of inquiry at the national level. to Treatment Response Our multidisciplinary Sarcoma Program continues to investigate better treatments for this rare cancer while providing patients with a level of expertise matched by few centers in the United States (p. 16). Our work on potential new therapies for acute myeloid leukemia (p. 20) and breast cancer (p. 13) demonstrates the promising results of the continued pursuit of inquiry for our patients. Finally, we demonstrate our ability to ask complex questions with the work we’re pursuing on laser interstitial therapy with colleagues in the Rose Ella Burkhardt Brain Tumor and Neuro-Oncology Center (p.
    [Show full text]
  • Download Issue
    Cell Circuitry || Science Teaches English || The Chicken Genome Is Hot || Magnets in Medicine SEPTEMBER 2002 www.hhmi.org/bulletin Leading Doublea Life It’s a stretch, but doctors who work bench to bedside say they wouldn’t do it any other way. FEATURES 14 On Human Terms 24 The Evolutionary War A small—some say too small—group of Efforts to undermine evolution education have physician-scientists believes the best science evolved into a 21st-century marketing cam- requires patient contact. paign that relies on legal acumen, manipulation By Marlene Cimons of scientific literature and grassroots tactics. 20 Engineering the Cell By Trisha Gura Adam Arkin sees the cell as a mechanical system. He hopes to transform molecular 28 Call of the Wild biology into a kind of cellular engineering Could quirky, new animal models help scien- and in the process, learn how to move cells tists learn how to regenerate human limbs or from sickness to health. avert the debilitating effects of a stroke? By M. Mitchell Waldrop By Kathryn Brown 24 In front of a crowd of 1,500, Ohio’s Board of Education heard testimony on whether students should learn about intelligent design in science class. DEPARTMENTS 2 NOTA BENE 33 PERSPECTIVE ulletin Intelligent Design Is a Cop-Out 4 LETTERS September 2002 || Volume 15 Number 3 NEWS AND NOTES HHMI TRUSTEES PRESIDENT’S LETTER 5 JAMES A. BAKER, III, ESQ. 34 Senior Partner, Baker & Botts A Creative Influence In from the Fields ALEXANDER G. BEARN, M.D. Executive Officer, American Philosophical Society 35 Lost on the Tip of the Tongue Adjunct Professor, The Rockefeller University UP FRONT Professor Emeritus of Medicine, Cornell University Medical College 36 Biology by Numbers FRANK WILLIAM GAY 6 Follow the Songbird Former President and Chief Executive Officer, SUMMA Corporation JAMES H.
    [Show full text]
  • Meet the Faculty Candidates
    MEET THE FACULTY CANDIDATES Candidates are displayed in alphabetically by last name. Prospective employers are invited to attend and while no event pre-registration is required however they must be registered for the BMES 2018 Annual Meeting. A business card will be required to enter the event. COMPLETE DETAILED CANDIDATE INFORMATION AVAILABLE AT www.bmes.org/faculty. Specialty - Biomaterials Alessia Battigelli Woo-Sik Jang Sejin Son John Clegg Patrick Jurney Young Hye Song R. Cornelison Kevin McHugh Ryan Stowers Yonghui Ding Yifeng Peng Varadraj Vernekar Victor Hernandez-Gordillo Shantanu Pradhan Scott Wilson Marian Hettiaratchi Eiji Saito Yaoying Wu Era Jain Andrew Shoffstall Specialty - Biomechanics Adam Abraham Vince Fiore Panagiotis Mistriotis Edward Bonnevie Zeinab Hajjarian Simone Rossi Alexander Caulk Xiao Hu Alireza Yazdani Venkat Keshav Chivukula Heidi Kloefkorn Rana Zakerzadeh Jacopo Ferruzzi Yizeng Li Specialty - Biomedical Imaging Mahdi Bayat Chong Huang Katheryne Wilson Zhichao Fan Jingfei Liu Kihwan Han Alexandra Walsh Specialty - BioMEMS Jaehwan Jung Aniruddh Sarkar Mengxi Wu Specialty - Cardiovascular Engineering Reza Avaz Kristin French Zhenglun (Alan) Wei Specialty - Cellular Engineering Annie Bowles Kate Galloway Kuei-Chun Wang Alexander Buffone Laurel Hind Mahsa Dabagh Matthew Kutys See other side for more candidates Specialty - Device Engineering (Microfluidics, Electronics, Machine-Body interface) Taslim Al-Hilal Brian Johnson David Myers Jungil Choi Tae Jin Kim Max Villa Haishui Huang Jiannan Li Ying Wang Specialty
    [Show full text]
  • Andrew Goldstein, Ph.D., Is an Assistant Professor Of
    Andrew S. Goldstein, Ph.D. Biography: Andrew Goldstein, Ph.D., is an Assistant Professor of Molecular, Cell and Developmental Biology and Assistant Professor of Urology in the David Geffen School of Medicine at the University of California, Los Angeles. Dr. Goldstein is a member of the Broad Stem Cell Research Center and the Jonsson Comprehensive Cancer Center at UCLA. Dr. Goldstein majored in Biochemistry and Molecular Biology at Dartmouth College before receiving his Ph.D. in Molecular Biology under the mentorship of Dr. Owen Witte at UCLA. He went on to start his own research group as a Fellow of the Broad Stem Cell Research Center at UCLA in 2011 before joining the faculty as an Assistant Professor in 2016. Dr. Goldstein’s research has been supported by a Prostate Cancer Foundation Young Investigator Award, Department of Defense PCRP Idea Development Award and an American Cancer Society Research Scholar Grant, and his work is currently supported by an NIH/NCI R01. He has been awarded the 2018 American Cancer Society Giants of Science Hope Award, 2019 SBUR Young Investigator Award and 2019 Rose Hills Innovator Award. At UCLA, Dr. Goldstein serves on committees for the Jonsson Comprehensive Cancer Center, the SPORE in Prostate Cancer, and the graduate program in Cell and Developmental Biology. He joined the Society for Basic Urologic Research in 2019 and currently participates as part of the membership committee (2020-present). Research Interests: Research in Dr. Goldstein’s laboratory is focused on defining factors that regulate prostate progenitor cell aging and transformation with an emerging interest in prostate epithelial metabolism.
    [Show full text]
  • My Name Is Michael Mark Gottesman and My Position Is Deputy Director for Intramural Research at the National Institutes of Health
    NHGRI: OH_Gottesman_Michael_20111113 1 3/1/16 My name is Michael Mark Gottesman and my position is deputy director for intramural research at the National Institutes of Health. I was born on October 7, 1946 in Jersey City, New Jersey. And when I was around two years old, my family moved to Flushing, Queens, and I had most of my formative years growing up in Flushing. I cannot remember a time when I wasn’t interested in science. Probably the first interaction with issues related to public health was as one of many probably millions of children in the United States who got the Salk vaccine as a -- as a test. I remember lining up, they explained to us that this was a trial, and we all got shots, which was not that much fun for a six-year-old or a seven-year-old. And that was a huge sea change. I remember learning about the fact that before then people got polio, kids got polio. They wandered off to camp, they came back paralyzed. And after that period, we didn’t need to worry about polio. So I had the sense that there was a lot that biomedical research could do to alleviate human disease. The next big event scientifically in my life was the launch of Sputnik in 1957, and it was a wake-up call to the United States. We were so-called “falling behind” in the space race, and I was an eleven-year-old boy who was interested in space science. So I spent my childhood after that making rockets, probably not as safely as it should have been, but no unfortunate accidents befell me.
    [Show full text]
  • Medical Advisory Board September 1, 2006–August 31, 2007
    hoWard hughes medical iNstitute 2007 annual report What’s Next h o W ard hughes medical i 4000 oNes Bridge road chevy chase, marylaNd 20815-6789 www.hhmi.org N stitute 2007 a nn ual report What’s Next Letter from the president 2 The primary purpose and objective of the conversation: wiLLiam r. Lummis 6 Howard Hughes Medical Institute shall be the promotion of human knowledge within the CREDITS thiNkiNg field of the basic sciences (principally the field of like medical research and education) and the a scieNtist 8 effective application thereof for the benefit of mankind. Page 1 Page 25 Page 43 Page 50 seeiNg Illustration by Riccardo Vecchio Südhof: Paul Fetters; Fuchs: Janelia Farm lab: © Photography Neurotoxin (Brunger & Chapman): Page 3 Matthew Septimus; SCNT images: by Brad Feinknopf; First level of Rongsheng Jin and Axel Brunger; iN Bruce Weller Blake Porch and Chris Vargas/HHMI lab building: © Photography by Shadlen: Paul Fetters; Mouse Page 6 Page 26 Brad Feinknopf (Tsai): Li-Huei Tsai; Zoghbi: Agapito NeW Illustration by Riccardo Vecchio Arabidopsis: Laboratory of Joanne Page 44 Sanchez/Baylor College 14 Page 8 Chory; Chory: Courtesy of Salk Janelia Farm guest housing: © Jeff Page 51 Ways Illustration by Riccardo Vecchio Institute Goldberg/Esto; Dudman: Matthew Szostak: Mark Wilson; Evans: Fred Page 10 Page 27 Septimus; Lee: Oliver Wien; Greaves/PR Newswire, © HHMI; Mello: Erika Larsen; Hannon: Zack Rosenthal: Paul Fetters; Students: Leonardo: Paul Fetters; Riddiford: Steitz: Harold Shapiro; Lefkowitz: capacity Seckler/AP, © HHMI; Lowe: Zack Paul Fetters; Map: Reprinted by Paul Fetters; Truman: Paul Fetters Stewart Waller/PR Newswire, Seckler/AP, © HHMI permission from Macmillan Page 46 © HHMI for Page 12 Publishers, Ltd.: Nature vol.
    [Show full text]
  • Biology Distilled Minimize Boom-And-Bust Cycles of Species Outbreaks and Ecosystem Imbalances
    COMMENT BOOKS & ARTS “Serengeti Rules”), which he shows are appli- cable both to the restoration of ecosystems and to the management of the biosphere. The same rule may carry different names in different biological contexts. The double- negative logic rule, for instance, enables a given gene product to feed back to slow down its own synthesis. In an ecosystem the same rule, known as top-down regulation, NHPA/PHOTOSHOT VINCE BURTON/ applies when the abundance of a predator (such as lynx) limits the rise in the popu- lation of prey (such as snowshoe hares). This is why, in Yellowstone National Park in Wyoming, the reintroduction of wolves has resulted in non-intuitive changes in hydrology and forest cover: wolves prey on elk, which disproportionately feed on streamside willows and tree seedlings. It is also why ecologists can continue to manage the Serengeti, and have been able to ‘rebuild’ a functioning ecosystem from scratch in Gorongosa National Park, Mozambique. Carroll argues that the rules regulat- Interactions between predators and prey, such as lynxes and hares, can be modelled with biological rules. ing human bodily functions — which have improved medical care and driven drug dis- ECOLOGY covery — can be applied to ecosystems, to guide conservation and restoration, and to heal our ailing planet. His Serengeti Rules encapsulate the checks and balances that Biology distilled minimize boom-and-bust cycles of species outbreaks and ecosystem imbalances. Eco- Brian J. Enquist reflects on a blueprint to guide logical systems that are missing key regula- the recovery of life on Earth. tory players, such as predators, can collapse; if they are overtaken by organisms spread by human activities, such as the kudzu vine, a an biology become as predictive as the unification of biology ‘cancer-like’ growth of that species can result.
    [Show full text]
  • Mapping Our Genes—Genome Projects: How Big? How Fast?
    Mapping Our Genes—Genome Projects: How Big? How Fast? April 1988 NTIS order #PB88-212402 Recommended Citation: U.S. Congress, Office of Technology Assessment, Mapping Our Genes-The Genmne Projects.’ How Big, How Fast? OTA-BA-373 (Washington, DC: U.S. Government Printing Office, April 1988). Library of Congress Catalog Card Number 87-619898 For sale by the Superintendent of Documents U.S. Government Printing Office, Washington, DC 20402-9325 (order form can be found in the back of this report) Foreword For the past 2 years, scientific and technical journals in biology and medicine have extensively covered a debate about whether and how to determine the function and order of human genes on human chromosomes and when to determine the sequence of molecular building blocks that comprise DNA in those chromosomes. In 1987, these issues rose to become part of the public agenda. The debate involves science, technol- ogy, and politics. Congress is responsible for ‘(writing the rules” of what various Federal agencies do and for funding their work. This report surveys the points made so far in the debate, focusing on those that most directly influence the policy options facing the U.S. Congress, The House Committee on Energy and Commerce requested that OTA undertake the project. The House Committee on Science, Space, and Technology, the Senate Com- mittee on Labor and Human Resources, and the Senate Committee on Energy and Natu- ral Resources also asked OTA to address specific points of concern to them. Congres- sional interest focused on several issues: ● how to assess the rationales for conducting human genome projects, ● how to fund human genome projects (at what level and through which mech- anisms), ● how to coordinate the scientific and technical programs of the several Federal agencies and private interests already supporting various genome projects, and ● how to strike a balance regarding the impact of genome projects on international scientific cooperation and international economic competition in biotechnology.
    [Show full text]
  • UNIVERSITY of CALIFORNIA Los Angeles Gene Editing of Bruton's
    UNIVERSITY OF CALIFORNIA Los Angeles Gene Editing of Bruton’s Tyrosine Kinase for Treatment of X-Linked Agammaglobulinemia A dissertation submitted in partial satisfaction of the requirements for the degree Doctor of Philosophy in Molecular Biology by David Gray 2020 © Copyright by David Gray 2020 ABSTRACT OF THE DISSERTATION Gene Editing of Bruton’s Tyrosine Kinase for Treatment of X-Linked Agammaglobulinemia by David Gray Doctor of Philosophy in Molecular Biology University of California, Los Angeles, 2020 Professor Donald Barry Kohn, Chair X-Linked Agammaglobulinemia (XLA) is a primary immunodeficiency characterized by a lack of mature B lymphocytes and antibody production. Patients with XLA have loss of function mutations in the Bruton’s Tyrosine Kinase (BTK) gene. The standard of care for XLA is immunoglobulin supplementation, which has a profound effect on patient wellbeing and life expectancies. However, immunoglobulin supplementation requires frequent, expensive injections throughout a patient’s life and patients remain susceptible to certain recurring illnesses. The only permanent cure for XLA is an allogeneic hematopoietic stem cell (HSC) transplant, though it is rarely performed due to the associated risks. Gene therapy-based methods to replace or repair the BTK gene in autologous HSCs provide an alternative with the benefits of a permanent cure for XLA while circumventing much of the risk. While previous efforts to deliver a functional copy of the BTK gene using viral vector mediated gene transfer have shown promise, these vectors carry a risk of insertional oncogenesis ii which may not be tolerated for treatment of XLA. Instead, this dissertation lays a foundation for targeted integration of a functional copy of the BTK gene into HSCs using Cas9 endonuclease mediated gene editing to drastically reduce those risks.
    [Show full text]
  • Structure & Symmetry
    HAPPY HOLIDAYS ASBMB MEMBERS December 2008 Structure & Symmetry American Society for Biochemistry and Molecular Biology J\\PfliGifk\`ej n`k_*.#'''>=G$kX^^\[FI=Zcfe\j 8 $@C- 9 "@C- : ; >=G$kX^^\[Kil\FI=Zcfe\jXi\kiXej]\Zk\[`ekf?<B)0* Z\ccjXe[k_\kX^^\[gifk\`ejXi\m`jlXc`q\[[li`e^@C$- `e[lZ\[elZc\XikiXejcfZXk`feJK8K*#gXe\c8Xe[9 Xe[`e Ôcfgf[`XXe[jki\jjÔY\i]fidXk`fe8Zk`e#gXe\c:Xe[; % Kil\FI= Fi`>\e\jXclk\j >\efd\n`[\FI=Zcfe\j k_\>=Gg`fe\\ij ]fik_\`iEfY\c ]fikX^^\[gifk\`e\ogi\jj`fe Gi`q\XnXi[ ×:$k\id`eXckX^f]>=G ×J\hl\eZ\m\i`Ô\[Xe[^lXiXek\\[ ×<Xj`cpj_lkkc\[`ekf)'[\jk`eXk`fem\Zkfij ×KiXej]\Zk`fe$i\X[p1('l^gcXjd`[;E8 fi`^\e\%Zfd&fi] ORG-041-GFPTaggedAd_ASBMB_v7.indd 1 10/20/08 12:29:39 PM contents DECEMBER 2008 ON THE COVER: Captivated by the symmetry society news of molecular structure, Sung-Hou Kim has been a 2 From the Editor leader in revealing symmetry 3 President’s Message through his studies in crystallography and 5 Letters to the Editor structural genomics. 30 6 Washington Update 12 Retrospective: Anthony G. San Pietro FASEB releases new Breakthroughs in special interest Bioscience. 6 13 Science’s Role in Foreign Policy 14 ASBMB Round Table: Jim Wells and Mary Woolley 16 Keeping Women in Science 19 Grammar and Writing Tips 2009 meeting 20 The 2009 Fritz Lipmann Lectureship: Douglas C. Rees 21 The 2009 ASBMB Merck Award: John Kuriyan 22 The 2009 FASEB Excellence in Science Award: Susan Lindquist science focus 30 Sung-Hou Kim: Consummate Crystallographer departments 7 News from the Hill 10 Member Spotlight 23 Education and Training A leaky pipeline for women scientists.
    [Show full text]
  • Training the Next Generation of Biomedical Investigators in Glycosciences
    Downloaded from http://www.jci.org on May 23, 2016. http://dx.doi.org/10.1172/JCI85905 The Journal of Clinical Investigation PERSPECTIVE Training the next generation of biomedical investigators in glycosciences Peter Agre,1 Carolyn Bertozzi,2 Mina Bissell,3 Kevin P. Campbell,4 Richard D. Cummings,5 Umesh R. Desai,6 Mary Estes,7 Terence Flotte,8 Guy Fogleman,9 Fred Gage,10 David Ginsburg,11 Jeffrey I. Gordon,12 Gerald Hart,13 Vincent Hascall,14 Laura Kiessling,15 Stuart Kornfeld,16 John Lowe,17 John Magnani,18 Lara K. Mahal,19 Ruslan Medzhitov,20 Richard J. Roberts,21 Robert Sackstein,22 Rita Sarkar,23 Ronald Schnaar,24 Nancy Schwartz,25 Ajit Varki,26 David Walt,27 and Irving Weissman28 1Department of Molecular Microbiology and Immunology, Johns Hopkins Bloomberg School of Public Health, Johns Hopkins University, Baltimore, Maryland, USA. 2Department of Chemistry, Stanford University, Stanford, California, USA. 3Lawrence Berkeley National Laboratory, Division of Biological Systems and Engineering, Berkeley, California, USA. 4Howard Hughes Medical Institute, Departments of Molecular Physiology and Biophysics, Neurology, and Internal Medicine, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA. 5Department of Surgery, Harvard Medical School, Beth Israel Deaconess Medical Center, Boston, Massachusetts, USA. 6Department of Medicinal Chemistry and Institute for Structural Biology, Drug Discovery and Development, Virginia Commonwealth University, Richmond, Virginia, USA. 7Department of Molecular Virology and Microbiology, Baylor College of Medicine, Houston, Texas, USA. 8Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts, USA. 9Federation of American Societies for Experimental Biology (FASEB), Bethesda, Maryland, USA. 10Laboratory of Genetics LOG-G, The Salk Institute for Biological Studies, La Jolla, California, USA.
    [Show full text]
  • The Gene Wars: Science, Politics, and the Human Genome
    8 Early Skirmishes | N A COMMENTARY introducing the March 7, 1986, issue of Science, I. Renato Dulbecco, a Nobel laureate and president of the Salk Institute, made the startling assertion that progress in the War on Cancer would be speedier if geneticists were to sequence the human genome.1 For most biologists, Dulbecco's Science article was their first encounter with the idea of sequencing the human genome, and it provoked discussions in the laboratories of universities and research centers throughout the world. Dul- becco was not known as a crusader or self-promoter—quite the opposite— and so his proposal attained credence it would have lacked coming from a less esteemed source. Like Sinsheimer, Dulbecco came to the idea from a penchant for thinking big. His first public airing of the idea came at a gala Kennedy Center event, a meeting organized by the Italian embassy in Washington, D.C., on Columbus Day, 1985.2 The meeting included a section on U.S.-Italian cooperation in science, and Dulbecco was invited to give a presentation as one of the most eminent Italian biologists, familiar with science in both the United States and Italy. He was preparing a review paper on the genetic approach to cancer, and he decided that the occasion called for grand ideas. In thinking through the recent past and future directions of cancer research, he decided it could be greatly enriched by a single bold stroke—sequencing the human genome. This Washington meeting marked the beginning of the Italian genome program.3 Dulbecco later made the sequencing
    [Show full text]