Rna Interference Technology: From Basic Science To Drug Development

RNA INTERFERENCE TECHNOLOGY RNA Interference (RNAi) technology has rapidly become one of the key methods used in functional genomics. RNAi is used to block the expression of genes and create phenotypes that can potentially yield clues about the function of these genes. In the postgenomic era, the elucidation of the physiological function of genes has become the rate-limiting step in the quest to develop “gene-based drugs” and RNAi could potentially play a pivotal role in the validation of such novel drugs. In this cutting-edge overview, the basic concepts of RNAi biology are discussed, as well as the current and potential applications. Leading experts from both academia and industry have contributed to this invaluable reference for graduate students, postdocs, and researchers from academia wanting to initiate RNAi research in their own labs, as well as for those working in research and development in biotech and pharmaceutical companies who need to understand this emerging technology. Krishnarao Appasani is the Founder and Chief Executive Officer of GeneExpression Systems, a gene discovery company focusing on functional genomics in cancer research. RNA Interference Technology FROM BASIC SCIENCE TO DRUG DEVELOPMENT Edited by Krishnarao Appasani GeneExpression Systems, Inc. Forewords by Andrew Fire Stanford University, co-discoverer of RNAi and Marshall Nirenberg National Institutes of Health Winner of the Nobel Prize in Physiology or Medicine, 1968 cambridge university press Cambridge, New York, Melbourne, Madrid, Cape Town, Singapore, São Paulo Cambridge University Press The Edinburgh Building, Cambridge cb2 2ru, UK Published in the United States of America by Cambridge University Press, New York www.cambridge.org Information on this title: www.cambridge.org/9780521836777 © Cambridge University Press 2005 This book is in copyright. Subject to statutory exception and to the provision of relevant collective licensing agreements, no reproduction of any part may take place without the written permission of Cambridge University Press. First published in print format isbn-13 isbn-10 978-0-511-08221-4 eBook (NetLibrary) 0-511-08221-5 eBook (NetLibrary) isbn-13 isbn-10 978-0-521-83677-7 hardback 0-521-83677-8 hardback Cambridge University Press has no responsibility for the persistence or accuracy of urls for external or third-party internet websites referred to in this book, and does not guarantee that any content on such websites is, or will remain, accurate or appropriate. In memory of my parents For my teachers, family members and especially my wife Shyamala and sons Raakish and Raghu Contents Foreword by Andrew Fire Foreword by Marshall Nirenberg