Gene Therapy for Neurological Disorders Methods and Protocols
This volume provides a clear and detailed roadmap of how to design and execute a gene therapy experiment in order to obtain consistent results. Chapters in this book disseminate bits of unknown information that are important to consider during the course
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Fredric P. Manfredsson Editor
Gene Therapy for Neurological Disorders Methods and Protocols
METHODS
IN
MOLECULAR BIOLOGY
Series Editor John M. Walker School of Life and Medical Sciences University of Hertfordshire Hatfield, Hertfordshire, AL10 9AB, UK
For further volumes: http://www.springer.com/series/7651
Gene Therapy for Neurological Disorders Methods and Protocols
Edited by
Fredric P. Manfredsson Translational Science and Molecular Medicine, Michigan State University, College of Human Science, Grand Rapids, Michigan, USA
Editor Fredric P. Manfredsson Translational Science and Molecular Medicine Michigan State University, College of Human Science Grand Rapids, Michigan, USA
ISSN 1064-3745 ISSN 1940-6029 (electronic) Methods in Molecular Biology ISBN 978-1-4939-3270-2 ISBN 978-1-4939-3271-9 (eBook) DOI 10.1007/978-1-4939-3271-9 Library of Congress Control Number: 2015953099 Springer New York Heidelberg Dordrecht London © Springer Science+Business Media New York 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 Humana Press is a brand of Springer Springer Science+Business Media LLC New York is part of Springer Science+Business Media (www.springer.com)
Preface Gene therapy of the nervous system, a technique once utilized by a few select laboratories, is now a commonplace research tool used around the world. Not only is gene therapy a useful utility in treating and creating preclinical models, but this technology has also demonstrated success in the clinic, in terms of both safety and efficacy [1, 2]. Gene therapy is a valuable tool that is being increasingly utilized to model neurodegenerative disorders [3]. One reason for this is the inherent ability of gene therapy to control genetic expression in both a spatial and temporal manner. For example, using this precision of gene therapy to model neurodegenerative disorders enables researchers to overcome any developmental compensations that may occur with germ line manipulations [4,
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