Stem Cell Biology and Gene Therapy - Peter J. Quesenberry.pdf

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Stem Cell Biology and Gene Therapy
Edited by Peter J. Quesenberry, Gary S. Stein, Bernard G. Forget, Sherman M. Weissman
Copyright 1998 Wiley-Liss, Inc.
ISBNs: 0-471-14656-0 (Paper); 0-471-22395-6 (Electronic)
STEM CELL BIOLOGY
AND GENE THERAPY
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STEMCELLBIOLOGY
ANDGENETHERAPY
Edited by
PETER J. QUESENBERRY
GARY S. STEIN
University of Massachusetts
Worcester, Massachusetts
BERNARD G. FORGET
SHERMAN M. WEISSMAN
Yale University School of Medicine
New Haven, Connecticut
A JOHN WILEY & SONS, INC., PUBLICATION
New York l Chichester l Weinheim l Brisbane l Singapore l Toronto
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ISBN 0-471-22395-6
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CONTENTS
Preface
vii
Contributors
ix
Stem Cell Systems: Basic Principles and Methodologies
Susan K. Nilsson and Peter J. Quesenberry
1
Cytokine/Growth Factor Responsivenessof Early Hemopoietic
Progenitor Cells
Antony W. Burgess
15
Molecular Mechanisms Controlling the Cell Cycle and
Proliferation-Differentiation Interrelationships
Gary S. Stein, Andre J. Van Wijnen, Dennet R. Hushka,
Baruch Frenkel, Jane B. Lian, and Janet L. Stein
41
Stem Cell Transcription
Sherman M. Weissman and Archibald S. Perkins
81
Hematopoietic Stem Cells: Proliferation, Purification and
Clinical Applications
Ruth Pettengell
133
and Malcolm
A. S. Moore
Delivery Systems for Gene Therapy: The Adenovirus
Thomas Shenk
161
Gene Transfer to Muscle and Spinal Cord Using Herpes
Simplex Virus-Based Vectors
Johnny
179
Huard,
William F. Goins, Giridhar
R. Akkaraju,
David Krisky,
Tom Oligino,
Peggy Marconi,
Charles S. Day, andJoseph
C. Glorioso
V
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vi
CONTENTS
8
Herpes Virus Vectors
201
Xandra 0. Breakefield, Peter Pechan, Karen Johnston,
and David Jacoby
9
Delivery Systems for Gene Therapy: Adeno-Associated Virus
Gabriele Kroner- Lux, Christopher E. Walsh,
and Richard Jude Sam&ski
235
10
Delivery Systems for Gene Therapy: Adeno-Associated
Virus 2
A run Srivastava
257
11
Ribozyme Gene Therapy Targeting Stem Cells for
Human Immunodeficiency Virus Infection
Anthony D. Ho, Ping Law, Xinqiang Li, and Flossie Wong-Staal
289
12
Elements of DNA Vaccine Design
Michael J. Caufield and Margaret A. Liu
309
13
Development of Gene Therapy for Gaucher Disease
J.A. Barranger, E. 0. Rice, J. Dun&an, M. Eljanne, N. Takivama,
M. Nimgaonkar, J. Mierski, M. Beeler, A. Kemp, J. Lancia,dS. Lucot,
S. Schierer-Fochler, J. Mannion-Henderson,
331
T. Mohney, W. Swaney,
A. Bahnson, V. Bansal, and E. Ball
14
Clinical Applications of Gene Therapy: Correction of Genetic
Disease Affecting Hematopoietic Cells
Jeffrev A. Medin, Johan Richter, and Stefan Karlsson
363
d
Gene Therapy for Hemophilia
Katherine A. High
15
385
16
Clinical Applications of Gene Therapy: Anemias
George F. Atweh and Bernard G. Forget
411
17
Clinical Applications of Gene Therapy in Cancer: Modification
of Sensitivity to Therapeutic Agents
Thomas Licht, Michael M. Gottesman, and Ira Pastan
429
18
Clinical Applications of Gene Therapy: Brian Tumors
Kenneth W. Culver and John C. Van Gilder
455
19
Clinical Applications of Gene Therapy: Cardiovascular Disease
Jonathan C. Fox
471
20
Applications of Gene Therapy to Neurological Diseases
and Injuries
Derek L. Choi-Lundberg
503
and Martha C. Bohn
Index
555
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