Gene Therapy: Treatments and Cures for Genetic Diseases

by Panno, Joseph

• ISBN13: 9780816068500
• ISBN10: 081606850X
• Edition: Revised
• Format: Hardcover
• Copyright: 2010-09-01
• Publisher: Facts on File

Summary

The New Biology is a 10-volume set has evolved to accommodate new material and new areas of research, in keeping with the expansion of the biological disciplines and the development and application of research technologies in and out of the scientific laboratory. Since its publication several years ago, the set now includes four new volumes that focus on issues that cover the spectrum of human health, maintenance, and stability.

Author Biography

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

Table of Contents

Preface	p. ix
Acknowledgments	p. xiii
Introduction	p. xiv
The Human Genome	p. 1
Historical Background	p. 2
The Human Genome Project	p. 11
Mobile Genetic Elements	p. 18
Gene Mutations	p. 20
Summary	p. 24
Genetic Disorders	p. 25
Cancer	p. 27
Cardiovascular Disease	p. 34
Cystic Fibrosis	p. 35
Hemophilia	p. 36
Immune Deficiencies	p. 39
Liver Disease	p. 40
Muscular Dystrophy	p. 41
Neurological Disorders	p. 42
Vision Impairment	p. 47
Viruses: Cornerstones off Gene Therapy	p. 51
Viruses Are Living Crystals	p. 52
Viral Genomes May Be RNA or DNA	p. 54
Viruses Evolved from Plasmids	p. 58
Viruses Know How to Infect Cells	p. 61
The Virus as a Gene Vehicle	p. 66
Viruses Used in Gene Therapy	p. 67
Viral Ecology	p. 70
Ashi DeSilva: A Promising Start	p. 72
Adenosine Deaminase	p. 75
Cells of the Immune System	p. 75
Preliminary Research	p. 80
Understanding Clinical Trials	p. 83
Medical Procedure for ADA Gene Therapy	p. 85
The DeSilva Clinical Trial	p. 86
Jesse Gelsinger: The First Fatality	p. 88
Ornithine Transcarbamylase	p. 91
Preliminary Research	p. 92
Medical Procedure for OTC Gene Therapy	p. 94
The Gelsinger Clinical Trial	p. 96
The Investigation	p. 98
Conclusions	p. 100
Future Prospects	p. 102
Safer Vehicles	p. 103
Reducing Immune Rejection of the Vector	p. 108
Improved Risk Assessment	p. 111
Redesigning Humans	p. 112
Ethical Issues	p. 121
The Belmont Report	p. 121
Biomedical Research	p. 125
Physiological Enhancement	p. 127
Cosmetic Applications	p. 128
Legal Issues	p. 130
Regulatory Agencies	p. 131
The Gelsinger Legal Trial	p. 135
International Regulation	p. 140
Clinical Trials	p. 142
Cancer	p. 142
Cystic Fibrosis	p. 144
Immunological Disorders	p. 147
Neurological Disorders	p. 150
Retinal Dystrophy	p. 153
Resource Center	p. 154
Cell Biology	p. 154
Biotechnology	p. 176
Gene and Protein Nomenclature	p. 185
Weights and Measures	p. 186
Glossary	p. 187
Further Resources	p. 216
Index	p. 225
Table of Contents provided by Ingram. All Rights Reserved.

Excerpts

The number of disorders treated with gene therapy has increased from a few in 1990 to more than 900 in 2009. Of all the technologies provided by the new biology, gene therapy holds the promise of unlimited potential for curing disease and reversing the effects of aging.
Gene Therapy, Revised Edition discusses the science behind gene therapy, as well as the ethical and legal issues associated with this therapy. Featuring updated and revised material—including many new full-color figures and photographs—this timely resource introduces two new chapters that focus on genomic research and the many recent gene therapy clinical trials. Additional genetic diseases are also highlighted, including retinal dystrophy, a form of blindness that is now treated with gene therapy in a clinical trial. Written in clear and accessible language, Gene Therapy, Revised Edition covers cell biology, biotechnology, and other topics relevant to gene therapy.