An Introduction to Molecular Medicine and Gene Therapy

Thomas F. Kresina0471391883, 9780471391883, 9780471461043

An Introduction to Molecular Medicine and Gene Therapy Edited by Thomas F. Kresina, Ph.D. Gene therapy, or the use of genetic manipulation for disease treatment, is derived from advances in genetics, molecular biology, clinical medicine, and human genomics. Molecular medicine, the application of molecular biological techniques to disease treatment and diagnosis, is derived from the development of human organ transplantation, pharmacotherapy, and elucidation of the human genome. An Introduction to Molecular Medicine and Gene Therapy provides a basis for interpreting new clinical and basic research findings in the areas of cloning, gene transfer, and targeting; the applications of genetic medicine to clinical conditions; ethics and governmental regulations; and the burgeoning fields of genomics, biotechnology, and bioinformatics. By dividing the material into three sections – an introduction to basic science, a review of clinical applications, and a discussion of the evolving issues related to gene therapy and molecular medicine-this comprehensive manual describes the basic approaches to the broad range of actual and potential genetic-based therapies. In addition, An Introduction to Molecular Medicine and Gene Therapy: * Covers new frontiers in gene therapy, animal models, vectors, gene targeting, and ethical/legal considerations * Provides organ-based reviews of current studies in gene therapy for monogenetic, multifactoral or polygenic disorders, and infectious diseases * Includes bold-faced terms, key concepts, summaries, and lists of helpful references by subject in each chapter * Contains appendices on commercial implications and a review of the history of gene therapy This textbook offers a clear, concise writing style, drawing upon the expertise of the authors, all renowned researchers in their respective specialties of molecular medicine. Researchers in genetics and molecular medicine will all find An Introduction to Molecular Medicine and Gene Therapy to be an essential guide to the rapidly evolving field of gene therapy and its applications in molecular medicine.

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Table of contents :
COVER……Page 1
CONTENTS……Page 6
PREFACE……Page 11
CONTRIBUTORS……Page 13
INDEX……Page 15
CHAPTER 1. Molecular Medicine and Gene Therapy: An Introduction……Page 31
CHAPTER 2. Nuclear Transplantation and New Frontiers in Genetic Molecular Medicine……Page 55
CHAPTER 3. Building a Better Mouse: Genetically Altered Mice as Models for Gene Therapy……Page 76
CHAPTER 4. Vectors of Gene Therapy……Page 105
CHAPTER 5. Gene Targeting……Page 141
CHAPTER 6. Gene Therapy for Hematological Disorders……Page 160
CHAPTER 7. Gene Therapy for Liver Disease……Page 180
CHAPTER 8. Gene Therapy in Cardiovascular Disease……Page 209
CHAPTER 9. Components of Cell and Gene Therapy for Neurological Disorders……Page 228
CHAPTER 10. Gene Therapy in the Treatment of Cancer……Page 259
CHAPTER 11. Gene Therapy for HIV Infection……Page 287
CHAPTER 12. Gene Therapy Approaches for Rheumatoid Arthritis……Page 315
CHAPTER 13. Federal Oversight of Gene Therapy Research……Page 326
CHAPTER 14. Ethical Issues in Molecular Medicine and Gene Therapy……Page 341
CHAPTER 15. Epilogue: Personal Genetic Medicine—The Future Is Now……Page 369
APPENDIX. Commercial Implications……Page 374