Somatic gene therapy

314 Pages·2018·33.54 MB·English

by  Chang

SOMATIC
GENE
THERAPY

SOMATIC
GENE
THERAPY
Edited by
Patricia
Boca Raton London New York
CRC PressC isR anC im Pprrinets osf, thIenc.
Taylor B&o Fcraanc Ris Gartoounp,, a nF ilnoforrimdaa business
First published 1995 by CRC Press
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Reissued 2018 by CRC Press
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Library of Congress Cataloging-in-Publication Data
Somatic gene therapy / edited by Patricia L. Chang
p. cm.
Includes bibliographical references and index.
ISBN 0-8493-2440-8
1. Gene Therapy.
[DNLM: 1. Gene Therapy. QZ 50 S693 1994]
RB155.8.S56 1994
616’ .042—dc20
DNLM/DLC
for Library of Congress 94-17728
A Library of Congress record exists under LC control number: 94017728
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been unable to contact.
ISBN 13: 978-1-315-89766-0 (hbk)
ISBN 13: 978-1-351-07676-0 (ebk)
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PREFACE
After a long gestation, with assistance from a large number of prenatal attendants,
gene therapy was born in September 1990. In honor of its fourth birthday, I have been
asked to write a preface for the "Baby Book" (though I wonder whether the editor and
publishers would think of Somatic Gene Therapy in quite this way).
How is the baby doing? As this volume richly documents, gene therapy is growing
at a spectacular rate. Today there are over 100 approved clinical protocols worldwide;
approximately two thirds of the protocols are directed at cancer, while the remainder
cover primarily genetic disease and AIDS.
However, the approved clinical protocols are only the tip of the iceberg. Gene
therapy requires extensive studies that range throughout the research/development
spectrum. Clinical protocols are supported by a broad-based pyramid of fundamental
research which includes molecular genetics, virology, and animal studies. In order to
encompass the full expanse of gene therapy, this volume is organized around target
organs and the leaders who helped pioneer each of these areas have authored the relevant
chapters. Although a number of books have been published about human gene therapy,
Somatic Gene Therapy is a truly comprehensive volume that documents progress on
essentially all the major fronts.
So, at four years of age the baby is doing extraordinarily well. Could such rapid
growth have been predicted? Maybe by somebody, but certainly not by me. Having
barely survived the immense skepticism, and even hostility, that surrounded our efforts
to bring about the birth, I would never have anticipated that a volume as sophisticated
as Somatic Gene Therapy would have been possible by year four.
It is with great pleasure and personal pride that I contribute this brief preface to our
baby's book. Congratulations to the editor and all the authors, not only for their efforts
in producing this fine volume, but also for their research successes that have helped
make gene therapy very much alive and well!
W. French Anderson, M.D.

THE EDITOR
Patricia L. Chang, Ph.D., is a Professor of Pediatrics in the Faculty of Health
Sciences at McMaster University in Hamilton, Ontario, Canada.
Dr. Chang received her B.Sc. degree from the University of Hong Kong in 1967 and
obtained her Ph.D. degree in 1971 from the Department of Biochemistry, University of
Western Ontario, Canada. After doing post -doctoral work at University of Waterloo and
Hospital for Sick Children, she was appointed an Assistant Professor at McMaster
University in 1979, then Associate Professor in 1984, and Professor since 1990. She is
also an associate member of the departments of Biology and Biomedical Sciences at
McMaster University.
Dr. Chang is a member of the American Association for the Advancement of
Science, American Society of Human Genetics, American Society of Biological Chem-
ists, Association for Women in Science, Society of Chinese Bioscientists in American
and Canadian Societies of Biochemistry and Genetics. She has been the recipient of a
March of Dimes Birth Defect Foundation Basil O'Connor Award, a Velleman and
Ontario Mental Health Scholarship, Medical Research Council of Canada Visiting
Scientist award, and a National Institute of Health Visiting Scientist award. She has also
been awarded research grants from the Medical Research Council of Canada, the March
of Dimes Birth Defect Foundation, Natural Sciences and Engineering Council of Canada,
Canadian Hemophilia Society, and Ontario Mental Health Foundation. Her current
research interests are in human gene therapy and molecular genetics of neurological
diseases.
CONTRIBUTORS
Julie K. Andersen Fred H. Gage
Division of Neurogerontology Department of Neurosciences
Andrus Gerontology Center University of California, San Diego
University of Southern California La Jolla, CA
Los Angeles, CA
Michael W. Heartlein
Brian J, Aneskievich Department of Molecular Biology
Division of Plastic Surgery Transkaryotic Therapies, Inc.
Robert Wood Johnson Medical School Cambridge, MA
University of Medicine and Dentistry of
New Jersey
Roland Jurecic
New Brunswick, NJ
Department of Molecular and Human
Genetics
John W. Belmont
Baylor College of Medicine
Department of Molecular and Human
Houston, TX
Genetics
Baylor College of Medicine
Houston, TX Donald B. Kohn
Division of Research Immunology/Bone
Malcolm K. Brenner Marrow Transplantation
Division of Bone Marrow Children's Hospital Los Angeles
Transplantation Los Angeles, CA
Department of Hematology-Oncology
St. Jude Children's Research Hospital Kotoku Kurachi
Memphis, TN Department of Human Genetics
University of Michigan Medical School
Kenneth W. Culver Ann Arbor, MI
Human Gene Therapy Research Institute
Iowa Methodist Medical Center
Fred D. Ledley
Des Moines, lA
GENEMEDICINE INC.
Houston, TX
Lisa J. Fisher
Department of Neurosciences
Anthony G. Letai
University of California, San Diego
Pritzker School of Medicine
La Jolla, CA
University of Chicago
Chicago, IL
Elaine Fuchs
Department of Molecular Genetics and
Cell Biology Louis M. Messina
Howard Hughes Medical Institute Department of Surgery
University of Chicago Section of Vascular Surgery
Chicago, IL University of Michigan
Ann Arbor, MI
Jasodhara Ray Hideaki Tahara
Department of Neurosciences Department of Surgery
University of California, San Diego University of Pittsburgh
La Jolla, CA Pittsburgh, PA
Richard F Selden Douglas A. Treco
Transkaryotic Therapies, Inc. Transkaryotic Therapies, Inc.
Cambridge, MA Cambridge, MA
James C. Stanley Kenneth I. Weinberg
Department of Surgery Division of Research Immunology/
Section of Vascular Surgery Bone Marrow Transplantation
University of Michigan Children's Hospital Los Angeles
Ann Arbor, MI Los Angeles, CA
Shou-Nan Yao
Department of Human Genetics
University of Michigan Medical School
Ann Arbor, MI