GENE THERAPY Recent Titles in Health and Medical Issues Today

GENE THERAPY Recent Titles in Health and Medical Issues Today Obesity Evelyn B. Kelly Stem Cells Evelyn B. Kelly Organ Transplantation David Petechuk Alternative Medicine Christine A. Larson GENE THERAPY Evelyn B. Kelly Health and Medical Issues Today GREENWOOD PRESS Westport, Connecticut ● London Library of Congress Cataloging-in-Publication Data Kelly, Evelyn B. Gene therapy / Evelyn B. Kelly. p. cm. — (Health and medical issues today ISSN 1558–7592) Includes bibliographical references and index. ISBN 978–0–313–33760–4 (alk. paper) 1. Gene therapy—Popular works. 2. Gene therapy—Moral and ethical aspects— Popular works. I. Title. RB155.8.K45 2007 616'.042—dc22 2007016143 British Library Cataloguing in Publication Data is available. Copyright © 2007 by Evelyn B. Kelly All rights reserved. No portion of this book may be reproduced, by any process or technique, without the express written consent of the publisher. Library of Congress Catalog Card Number: 2007016143 ISBN: 978–0–313–33760–4 ISSN: 1558–7592 First published in 2007 Greenwood Press, 88 Post Road West, Westport, CT 06881 An imprint of Greenwood Publishing Group, Inc. www.greenwood.com Printed in the United States of America The paper used in this book complies with the Permanent Paper Standard issued by the National Information Standards Organization (Z39.48–1984). 10987654321 CONTENTS Series Foreword vii Preface ix Section One Scientific Background of Gene Therapy 1 Gene Therapy: A Treatment for Altered Genes 3 2 Medical and Scientific Issues in Gene Therapy 13 3 Discoveries Leading to Gene Therapy 25 4 Successes and Setbacks in the 1990s 35 5 Single-Gene Recessive Disorders 43 6 Single-Gene Traits: Dominant Disorders 55 7 X-Linked Disorders 63 8 Multigene and Traits Influenced by the Environment 73 Section Two Ethics and Regulations 9 Ethics Issues and Gene Therapy 91 10 Regulatory Actions in the United States 97 vi CONTENTS 11 Gene Therapy in Other Countries 105 12 Social and Religious Issues 111 13 The Future of Gene Therapy 119 Section Three References and Resources A Annotated Primary Source Documents 129 BTimeline for the Advance of Gene Therapy 181 C Further Reading 187 D Glossary 193 Index 197 SERIES FOREWORD Every day, the public is bombarded with information on developments in medicine and health care. Whether it is on the latest techniques in treatments or research, or on concerns over public health threats, this information directly impacts the lives of people more than almost any other issue. Although there are many sources for understanding these topics—from Web sites and blogs to news- papers and magazines—students and ordinary citizens often need one resource that makes sense of the complex health and medical issues affecting their daily lives. The Health and Medical Issues Today series provides just such a one-stop resource for obtaining a solid overview of the most controversial areas of health care today. Each volume addresses one topic and provides a balanced summary of what is known. These volumes provide an excellent first step for students and lay people interested in understanding how health care works in our society today. Each volume is broken into several sections to provide readers and researchers with easy access to the information they need: • Section I provides overview chapters on background information—including chapters on such areas as the historical, scientific, medical, social, and legal issues involved—that a citizen needs to intelligently understand the topic. • Section II provides capsule examinations of the most heated contemporary issues and debates, and analyzes in a balanced manner the viewpoints held by various advocates in the debates. viii SERIES FOREWORD • Section III provides a selection of reference material, including annotated primary source documents, a timeline of important events, and an annotated bibliography of useful print and electronic resources that serve as the best next step in learning about the topic at hand. The Health and Medical Issues Today odayTalthandMedicalIssuesseries strives to provide readers with all the information needed to begin making sense of some of the most important debates going on in the world today. The series will include volumes on such topics as stem-cell research, obesity, gene therapy, alternative medicine, organ transplantation, mental health, and more. PREFACE The popular Teenage Mutant Ninja Turtles introduced the word mutant into everyday language. Since publication of the first Turtles comic book in 1984, thousands of young people and adults have gasped and laughed as the powerful Turtles slashed their way through life with little concern for the reality of what a mutant is. The fact that a mutant is a gene that has changed, and the fact that the changed characteristic may be passed on to successive generations, has been totally unimportant to Turtles fans. In fact, people throughout history—not knowing about genes and mutation— have not cared about the diseases that people are born with. Disorders and dis- abilities were considered simply the result of fate. Infectious diseases became the scourge of the human race when such pathogens as bubonic plague, typhoid, and typhus killed large segments of the population. However, the advent of public health programs, immunizations, and antibiotics has helped conquer most of these microbes. Scientists in the latter half of the twentieth century were consumed with the developing science of genetics and with understanding how people inherit cer- tain characteristics. Advances in molecular biology triggered remarkable expan- sion in the knowledge of human genetics and the understanding of how genes gone awry could cause diseases and disorders. The next step was logical. Some scientists in the mid-1980s began to toy with the hypothesis that if a gene causes a disease, then it should be possible to cure the disease by removing the “bad” gene and replacing it with a “good” gene. Gene therapy seemed to make sense as a logical and straightforward solution to the scourge of genetic disease. In reality, the problem is much more complex. x PREFACE Misunderstanding and lack of knowledge color public perception of gene therapy. Actually, there is no one single kind of gene therapy, but many kinds of ther- apies dealing with different targets. As one scientist observed, “Gene therapy is not gene therapy is not gene therapy.” Although there have been setbacks and public relations snafus, researchers are forging ahead in the belief that gene therapy is a viable and workable technique. However, the rise of these new genetic technologies evokes concern among religious, scientific, and civic leaders that research geneticists’ immense power may spin out of control. This book will attempt to clarify what gene therapy is and the issues related to it. The work is organized in three sections according to the plan of Greenwood Press’s Health and Medical Issues Today series. Section One (Chapters 1 through 8) presents basic information for understanding gene therapy issues. Chapter 1 presents an overview, with background information about genetics and proteins. Chapter 2 relates medical and scientific information necessary for understanding gene therapy. Chapter 3 traces the history of genes as hereditary vehicles, and details how people began to accept the idea of exchanging bad genes for good genes. Chapter 4 traces the successes and setbacks of the 1990s and brings research topics into the twenty-first century. Chapters 5 through 8 discuss specific diseases and conditions that are targets for gene therapy. The chapters are arranged according to the patterns of genetic inheritance: single-gene recessive, single-gene dominant, X-linked conditions, and multigene conditions. Section Two (Chapters 9 through 13) covers issues related to gene therapy, ranging from the argumentation of ethical questions to discussion of regulatory efforts in the United States, gene therapy developments in other countries, the social and religious perspectives, and the future of gene therapy. Section Three concludes the discussion with annotated primary sources, a timeline important for the understanding of gene therapy, a glossary, sources for further information, and an index. This book is intended as a reference for students and other interested readers, and attempts to describe medical and scientific concepts in common language. Nothing in the book is intended as a substitute for medical advice. For information about personal circumstances, consult a physician or other health care pro- fessional. Boldface type indicates the first use of key words, which are listed in the glossary at the end of the book. S ECTION O NE Scientific Background of Gene Therapy Section One, consisting of Chapters 1 through 8, presents the foundation of the science that establishes gene therapy research. This section considers the scientific background, the tools for gene therapy, the historical development of gene therapy, and the diseases and disorders targeted for research. CHAPTER 1 Gene Therapy: A Treatment for Altered Genes As car horns honk and taxis whiz by, the busy, crowded streets of New York City, Chicago, or Los Angeles are awhirl with bustling people—skinny and fat, light and dark, loud and quiet—scurrying like ants in all directions. Their genetics and inheritance have created the diverse ways these people look and behave. But even more startling is that each of these individuals is carrying in his or her genetic makeup about half a dozen defective, really “bad” genes. The carriers are proba- bly blissfully unaware of this fact—unless they or their close relatives are among the millions who have a genetic disease. Statisticians tell us that about one in ten people has or will develop a genetic disorder at some stage in life. In 1983 Victor McKusick, Professor of Medical Genetics at Johns Hopkins University, Baltimore, estimated that 2,000 to 3,000 genetic diseases can be traced to specific genes. Since that time, according to Access Excellence (1990), researchers have determined that about 2,800 of these diseases are caused by defects or mutations in just one of the patient’s genes.

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