Gene Therapy: Treating Disease by Repairing Genes

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Infobase Publishing, 2005 - Gene therapy - 193 pages
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Dealing with one of the most controversial aspects of the new biology, Gene Therapy explores the development and future of this area of research and explains why it is considered such a promising branch of science. This encyclopedia presents an overview of genetic defects, viruses, and gene therapy in a way that students and general readers will find easy to understand. Comprehensive in scope, this book provides an account of the research leading to the first successful gene therapy trial and discusses the worldwide attention and subsequent controversy caused by the tragic death of a patient receiving gene therapy, as well as the future prospects and general ethics of gene therapy. A list of further reading, a glossary, and an index round out this timely reference.
 

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Contents

1 Genetic Disorders
1
Breast Cancer
4
Colon Cancer
5
Cystic Fibrosis
6
Hemophilia
7
Liver Disease
9
Cardiovascular Disease
10
Muscular Dystrophy
11
Clinical Procedure for OTC Gene Therapy
49
The Gelsinger Clinical Trial
51
The Investigation
52
Concluding Remarks
54
5 Future Prospects
56
Improved Risk Assessment
63
Redesigning Human Anatomy and Physiology
65
6 Ethics of Gene Therapy
71

Parkinsons Disease
12
Huntingtons Disease
13
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
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
Down to Earth
45
Ornithine Transcarbamylase OTC
46
Preliminary Research
48
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
Recombinant DNA Primer
107
The Human Genome Project
118
XLinked Severe Combined Immunodeficiency SCIDX1
121
Alzheimers Disease AD
123
Huntingdons Disease HD
124
Glossary
125
Further Reading
151
Index
157
Copyright

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About the author (2005)

Joseph Panno, Ph.D., holds a degree in biology from Simon Fraser University, British Columbia, and specializes in molecular biology and physiology. He has taught a variety of undergraduate biology courses and has been published in numerous peer-reviewed publications including

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