Genome Editing The Next Step in Gene Therapy
This comprehensive volume explores human genetic engineering its pre-clinical and clinical applications, current developments, and as treatment for hereditary diseases. It presents and evaluates the most recent advances in the understanding of mammalian h
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Toni Cathomen Matthew Hirsch Matthew Porteus Editors
Genome Editing
The Next Step in Gene Therapy
Advances in Experimental Medicine and Biology Volume 895 Editorial Board: IRUN R. COHEN, The Weizmann Institute of Science, Rehovot, Israel ABEL LAJTHA, N.S. Kline Institute for Psychiatric Research, Orangeburg, NY, USA JOHN D. LAMBRIS, University of Pennsylvania, Philadelphia, PA, USA RODOLFO PAOLETTI, University of Milan, Milan, Italy
More information about this series at http://www.springer.com/series/5584
Toni Cathomen • Matthew Hirsch Matthew Porteus Editors
Genome Editing The Next Step in Gene Therapy
Editors Toni Cathomen Medical Center - University of Freiburg Freiburg, Germany
Matthew Hirsch University of North Carolina Chapel Hill, NC, USA
Matthew Porteus Stanford Medical School Palo Alto, CA, USA
ISSN 0065-2598 ISSN 2214-8019 (electronic) Advances in Experimental Medicine and Biology ISBN 978-1-4939-3507-9 ISBN 978-1-4939-3509-3 (eBook) DOI 10.1007/978-1-4939-3509-3 Library of Congress Control Number: 2015958888 Springer New York Heidelberg Dordrecht London © American Society of Gene and Cell Therapy 2016 This work is subject to copyright. All rights are reserved by the Publisher, whether the whole or part of the material is concerned, specifically the rights of translation, reprinting, reuse of illustrations, recitation, broadcasting, reproduction on microfilms or in any other physical way, and transmission or information storage and retrieval, electronic adaptation, computer software, or by similar or dissimilar methodology now known or hereafter developed. The use of general descriptive names, registered names, trademarks, service marks, 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. The publisher, the authors and the editors are safe to assume that the advice and information in this book are believed to be true and accurate at the date of publication. Neither the publisher nor the authors or the editors give a warranty, express or implied, with respect to the material contained herein or for any errors or omissions that may have been made. Printed on acid-free paper Springer Science+Business Media LLC New York is part of Springer Science+Business Media (www.springer.com)
This book is dedicated to the memory of Carlos F. Barbas III. Among his incredibly long list of accomplishments, he was best known in the field of genome engineering for his invaluable contributions in designing zinc-finger arrays that have been used in artificial transcription factors, zinc-finger nucleases, and custom recombinases. “It has been a dream of mine to develop drugs that make a difference” (Carlos Barbas, 2008)
Preface
In the early 1900s, human genetic engineering remained the fanciful speculation of science fiction writers, scientists, and philosophers. The surfaced ideas perceived consequences that, already more than 100 years ago, invoked ethical dilemmas
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