Gene Therapy / Edition 2

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More About This Textbook


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.

Gene Therapy Revised Edition is a compelling examination of this controversial form of treatment that faces close scrutiny by the public, the media, and the government. Scientists have identified several thousand genetic anomalies that are known to be responsible for diseases and disorders, including cancer, senility, diabetes, retinal dystrophy, and asthma. Gene therapy attempts to cure these diseases by replacing the damaged gene that is the cause of the problem. Over the past 20 years, the number of disorders treated with gene therapy has increased from a few to close to a thousand. The book is a timely resource that discusses the science behind gene therapy, as well as the ethical and legal issue associated with it.

The volume includes information on

Ashi DeSilva

clinical trials

ethical and legal issues and controversies

Jesse Gelsinger

genomic research

the human genome

"redesingning" humans


The book contains more than 50 color photographs and line illustrations, a Resource Center of supporting materials in cell biology and biotechnology, a glossary, a list of print and Internet resources, and an index. The New Biology set is essential for middle and high school students, teachers, and general readers who wish to understand the latest information on this controversial area of scientific research.

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Product Details

  • ISBN-13: 9780816068500
  • Publisher: Facts on File, Incorporated
  • Publication date: 10/1/2010
  • Series: The New Biology Series
  • Edition description: REV
  • Edition number: 2
  • Pages: 256
  • Age range: 11 - 17 Years
  • Product dimensions: 6.10 (w) x 9.10 (h) x 0.70 (d)

Table of Contents

Preface ix

Acknowledgments xiii

Introduction xiv

1 The Human Genome 1

Historical Background 2

The Human Genome Project 11

Mobile Genetic Elements 18

Gene Mutations 20

Summary 24

2 Genetic Disorders 25

Cancer 27

Cardiovascular Disease 34

Cystic Fibrosis 35

Hemophilia 36

Immune Deficiencies 39

Liver Disease 40

Muscular Dystrophy 41

Neurological Disorders 42

Vision Impairment 47

3 Viruses: Cornerstones off Gene Therapy 51

Viruses Are Living Crystals 52

Viral Genomes May Be RNA or DNA 54

Viruses Evolved from Plasmids 58

Viruses Know How to Infect Cells 61

The Virus as a Gene Vehicle 66

Viruses Used in Gene Therapy 67

Viral Ecology 70

4 Ashi DeSilva: A Promising Start 72

Adenosine Deaminase 75

Cells of the Immune System 75

Preliminary Research 80

Understanding Clinical Trials 83

Medical Procedure for ADA Gene Therapy 85

The DeSilva Clinical Trial 86

5 Jesse Gelsinger: The First Fatality 88

Ornithine Transcarbamylase 91

Preliminary Research 92

Medical Procedure for OTC Gene Therapy 94

The Gelsinger Clinical Trial 96

The Investigation 98

Conclusions 100

6 Future Prospects 102

Safer Vehicles 103

Reducing Immune Rejection of the Vector 108

Improved Risk Assessment 111

Redesigning Humans 112

7 Ethical Issues 121

The Belmont Report 121

Biomedical Research 125

Physiological Enhancement 127

Cosmetic Applications 128

8 Legal Issues 130

Regulatory Agencies 131

The Gelsinger Legal Trial 135

International Regulation 140

9 Clinical Trials 142

Cancer 142

Cystic Fibrosis 144

Immunological Disorders 147

Neurological Disorders 150

Retinal Dystrophy 153

10 Resource Center 154

Cell Biology 154

Biotechnology 176

Gene and Protein Nomenclature 185

Weights and Measures 186

Glossary 187

Further Resources 216

Index 225

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