Stem cell biology and gene therapy

Contributors Stem Cell Systems: Basic Principles and Methodologies Susan K. Nilsson and Peter J. Quesenberry Cytokine/Growth Factor Responsiveness of Early Hemopoietic Progenitor Cells Antony W. Burgess 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 Stem Cell Transcription Sherman M. Weissman and Archibald S. Perkins Hematopoietic Stem Cells: Proliferation, Purification and Clinical Applications Ruth Pettengell and Malcolm A. S. Moore Delivery Systems for Gene Therapy: The Adenovirus Thomas Shenk Gene Transfer to Muscle and Spinal Cord Using Herpes Simplex Virus-Based Vectors Johnny Huard, William F. Goins, Giridhar R. Akkaraju, David Krisky, Tom Oligino, Peggy Marconi, Charles S. Day, andJoseph C. Glorioso vii ix 1 15 41 81 133 161 179 V vi 8 9 10 11 12 13 14 15 16 17 18 19 20 CONTENTS Herpes Virus Vectors 201 Xandra 0. Breakefield, Peter Pechan, Karen Johnston, and David Jacoby Delivery Systems for Gene Therapy: Adeno-Associated Virus Gabriele Kroner- Lux, Christopher E. Walsh, and Richard Jude Sam&ski 235 Delivery Systems for Gene Therapy: Adeno-Associated Virus 2 A run Srivastava 257 Ribozyme Gene Therapy Targeting Stem Cells for Human Immunodeficiency Virus Infection Anthony D. Ho, Ping Law, Xinqiang Li, and Flossie Wong-Staal 289 Elements of DNA Vaccine Design Michael J. Caufield and Margaret A. Liu 309 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, T. Mohney, W. Swaney, A. Bahnson, V. Bansal, and E. Ball 331 Clinical Applications of Gene Therapy: Correction of Genetic Disease Affecting Hematopoietic Cells Jeffrevd A. Medin, Johan Richter, and Stefan Karlsson Gene Therapy for Hemophilia Katherine A. High 363 385 Clinical Applications of Gene Therapy: Anemias George F. Atweh and Bernard G. Forget Clinical Applications of Gene Therapy in Cancer: Modification of Sensitivity to Therapeutic Agents Thomas Licht, Michael M. Gottesman, and Ira Pastan 411 429 Clinical Applications of Gene Therapy: Brian Tumors Kenneth W. Culver and John C. Van Gilder Clinical Applications of Gene Therapy: Cardiovascular Disease Jonathan C. Fox Applications of Gene Therapy to Neurological Diseases and Injuries Derek L. Choi-Lundberg and Martha C. Bohn