Adeno-Associated Virus Methods and Protocols
Today, progress in rAAV-mediated gene transfer is so robust that long-term, efficient, and regulatable transgene expression is reproducibly achieved in large animal models. The complexity of gene transfer agents in the context of their clinical use
- PDF / 6,846,481 Bytes
- 468 Pages / 504.57 x 720 pts Page_size
- 20 Downloads / 198 Views
IN
MOLECULAR BIOLOGY™
Series Editor John M. Walker School of Life Sciences University of Hertfordshire Hatfield, Hertfordshire, AL10 9AB, UK
For further volumes: http://www.springer.com/series/7651
Adeno-Associated Virus Methods and Protocols Edited by
Richard O. Snyder University of Florida, Gainesville, FL, USA
Philippe Moullier Unité INSERM 649, Nantes, France
Editors Richard O. Snyder University of Florida Department of Molecular Genetics and Microbiology 1600 SW Archer Road Gainesville, FL 32610 USA [email protected]
Philippe Moullier CHU Hôtel Dieu INSERM U649 Labo. Thérapie Génique bd. Jean Monnet 30 44035 Nantes CX 1 Bâltiment Jean Monnet France [email protected]
ISSN 1064-3745 e-ISSN 1940-6029 ISBN 978-1-61779-369-1 e-ISBN 978-1-61779-370-7 DOI 10.1007/978-1-61779-370-7 Springer New York Dordrecht Heidelberg London Library of Congress Control Number: 2011939472 © Springer Science+Business Media, LLC 2011 All rights reserved. This work may not be translated or copied in whole or in part without the written permission of the publisher (Humana Press, c/o Springer Science+Business Media, LLC, 233 Spring Street, New York, NY 10013, USA), except for brief excerpts in connection with reviews or scholarly analysis. Use in connection with any form of information storage and retrieval, electronic adaptation, computer software, or by similar or dissimilar methodology now known or hereafter developed is forbidden. The use in this publication of trade names, trademarks, service marks, and similar terms, even if they are not identified as such, is not to be taken as an expression of opinion as to whether or not they are subject to proprietary rights. Printed on acid-free paper Humana Press is part of Springer Science+Business Media (www.springer.com)
Preface From a drug development perspective, gene transfer technology is relatively new and evolving at a rapid pace. Recombinant adeno-associated viral (rAAV) vectors have become more widely investigated and improved in their short history [1, 2]. Although wild-type AAV was studied for decades, the work of Xiao Xiao and R. Jude Samulski published in 1996 represents the first evidence that rAAV can be directly administered in situ resulting in efficient, remarkably tolerated, and long-term gene transfer in the mouse skeletal muscle following a single injection [3]. That year was also the year of the lentivirus vector capable of transducing resting neurons after intracerebral injection in the murine model [4]. Yet, 1996 was only 1 year after the Orkin and Motulsky report [5] emphasizing the need for better vectors. Today, progress in rAAV-mediated gene transfer is so robust that long-term, efficient, and regulatable transgene expression is reproducibly achieved in large animal models. For example, (1) the entire limb of hemophilia dogs and primates can be efficiently transduced resulting in long-term phenotypic correction [6, 7] and very recently in hemophilia B patients [8]; (2) rAAV administered once in nonhuman primate muscle shows sustained regulatable tr
Data Loading...
