Adeno-Associated Virus (AAV) Vectors in Gene Therapy
Human gene therapy holds great promise for the cure of many genetic diseases. In order to achieve such a cure there are two requirements. First, the affected gene must be cloned, its se quence determined and its regulation adequately characterized. Secon
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Editors R.W. Compans, Atlanta/Georgia M. Cooper, Birmingham/Alabama· H. Koprowski, Philadelphia/Pennsylvania· F. Melchers, Basel M. Oldstone, La Jolla/California· S. Olsnes, Oslo M. Potter, Bethesda/Maryland· H. Saedler, Cologne P.K. Vogt, La Jolla/California· H. Wagner, Munich
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Adeno-Associated Virus (AAV) Vectors in Gene Therapy
Edited by K.I. Berns and C. Giraud
With 38 Figures
,
Springer
Professor KENNETH I. BERNS, M.D., Ph.D. CATHERINE GIRAUD, Ph.D. Cornell University Medical College Hearst Microbiology Research Center Department of Microbiology 1300 York Avenue, Box 62 New York, NY 10021 USA
Cover illustration: The inverted terminal repeat of the adenoassociated virus genome is shown in the folded conformation which maximizes base pairing, The inverted terminal repeat plays a significant role in the regulation of viral gene expression, DNA replication, and integration into and excision from the integration site in the human genome. The red letters indicate the binding site of the viral regulatory protein "rep 68/78." "trs" indicates the site nicked by the rep 68/78 protein. Cover design: Design & Production, Heidelberg ISSN 0070-217X ISBN-13: 978-3-642-80209-6 e-ISBN-13: 978-3-642-80207-2 001: 10.1007/978-3-642-80207-2 This work is subject to copyright. All rights are reserved, whether the whole or part of the material is concerned, specifically the rights of translation, reprinting, reuse of illustrations, recitation, broadcasting, reproduction on microfilm or in any other way, and storage in data banks. Duplication of this publication or parts thereof is permitted only under the provisions of the German Copyright Law of September 9, 1965, in its current version, and permission for use must always be obtained from SpringerVerlag. Violations are liable for prosecution under the German Copyright Law.
© Springer-Verlag Berlin Heidelberg 1996 Softcover reprint of the hardcover 1st edition 1996 Library of Congress Catalog Card Number 15 - 12910 The use of general descriptive names, registered names, trademarks, etc. in this publication does not imply, even in the absence of a specific statement, that such names are exempt from the relevant protective laws and regulations and therefore free for general use. Product liability: The publishers cannot guarantee the accuracy of any information about dosage and application contained in this book. In every individual case the user must check such information by consulting other relevant literature. Typesetting: Scientific Publishing Services (P) Ltd, Madras SPIN: 10528856
27/3020/SPS - 5432 1 0 - Printed on acid-free paper
Preface
Human gene therapy holds great promise for the cure of many genetic diseases. In order to achieve such a cure there are two requirements. First, the affected gene must be cloned, its sequence determined and its regulation adequately characterized. Second, a suitable vector for the delivery of a good copy of the affected
