GENE THERAPY: reating Disease by Repairing Genes
GENE THERAPY reating Disease by Repairing Genes


The term new biologywas coined in the 1970s with the introduction of recombinant DNA technology (or biotechnology). At that time, biology was largely a descriptive science in danger of going adrift. Microbiologists at the turn of the century had found cures for a few diseases, and biologists in the 1960s had cracked the genetic code, but there was still no way to study the function of a gene or the cell as a whole. Biotechnology changed all that, and scientists of the period referred to it as the new technique or the new biology. However, since that time it has become clear that the advent of biotechnology was only the first step toward a new biology, a biology that now includes nuclear transfer technology (animal cloning), gene therapy, and stem cell therapy. All these technologies are covered in the six volumes of this set.

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Preface xi
Acknowledgments xiii
Introduction xv
1 Genetic Disorders 1
Immune Deficiencies 1
Breast Cancer 4
Colon Cancer 5
Melanoma 5
Cystic Fibrosis 6
Hemophilia 7
Liver Disease 9
Cardiovascular Disease 10
Muscular Dystrophy 11
Alzheimer’s Disease 11
Parkinson’s Disease 12
Huntington’s Disease 13
2 Viruses: The Cornerstone of Gene Therapy 14
Viruses Are Living Crystals 15
Viral Genomes May Be RNA or DNA 16
Viruses Evolved from Plasmids 22
Viruses Know How to Infect Cells 23
The Virus as a Gene Vehicle 28
Viruses Used in Gene Therapy 29
3 Ashi DeSilva: A Promising Start 32
Clinical Trials Defined 34
Cells of the Immune System 35
Adenosine Deaminase (ADA) 38
Preliminary Research 40
Clinical Procedure for ADA Gene Therapy 42
The DeSilva Clinical Trial 42
4 Jesse Gelsinger: Down to Earth 45
Ornithine Transcarbamylase (OTC) 46
Preliminary Research 48
Clinical Procedure for OTC Gene Therapy 49
The Gelsinger Clinical Trial 51
The Investigation 52
Concluding Remarks 54
5 Future Prospects 56
Safer Vehicles 56
Reducing Immune Rejection of the Vector 61
Improved Risk Assessment 63
Redesigning Human Anatomy and Physiology 65
6 Ethics of Gene Therapy 71
The Belmont Report 71
Clinical Trials 74
Physiological Enhancement 76
Cosmetic Applications 77
7 Legal Issues 79
Regulatory Agencies 80
The Gelsinger Legal Trial 83
International Regulation 86
8 Resource Center 87
Eukaryote Cell Primer 87
Recombinant DNA Primer 107
The Human Genome Project 118
X-Linked Severe Combined Immunodeficiency
(SCID-X1) 121
Alzheimer’s Disease (AD) 123
Huntingdon’s Disease (HD) 124
Glossary 125
Further Reading 151
Index 15

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