Translational Neuroscience Fundamental Approaches for Neurological D
Translational Neuroscience offers a far-reaching and insightful series of perspectives on the effort to bring potentially revolutionary new classes of therapies to the clinic, thereby transforming the treatment of human nervous system disorders. Great adv
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Translational Neuroscience Fundamental Approaches for Neurological Disorders
Translational Neuroscience
Mark H. Tuszynski Editor
Translational Neuroscience Fundamental Approaches for Neurological Disorders
Editor Mark H. Tuszynski Translational Neuroscience Institute University of California-San Diego Veterans Administration Medical Center La Jolla, CA, USA
ISBN 978-1-4899-7652-9 ISBN 978-1-4899-7654-3 DOI 10.1007/978-1-4899-7654-3
(eBook)
Library of Congress Control Number: 2015944984 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 Springer Science+Business Media LLC New York is part of Springer Science+Business Media (www.springer.com)
Contents
1
Introduction . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 1 Mark H. Tuszynski
Part I
Molecular Approaches
2
Gene Therapy of CNS Disorders Using Recombinant AAV Vectors . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 9 Giridhar Murlidharan, R. Jude Samulski, and Aravind Asokan
3
NGF and BDNF Gene Therapy for Alzheimer’s Disease . . . . . . . . . . . 33 Mark H. Tuszynski and Alan H. Nagahara
4
GDNF and AADC Gene Therapy for Parkinson’s Disease . . . . . . . . . . 65 Krystof Bankiewicz, Waldy San Sebastian, Lluis Samaranch, and John Forsayeth
5
GAD Gene Therapy for Parkinson’s Disease . . . . . . . . . . . . . . . . . . . . . 89 Michael G. Kaplitt and Matthew J. During
6
Antisense Oligonucleotides for Amyotrophic Lateral Sclerosis . . . . . . 99 Wade K. Self and Timothy M. Miller
7
Gene Therapy for Inborn Errors of Metabolism: Batten Disease . . . 111 Dolan Sondhi, Ronald G. Crystal, and Stephen M. Kaminsky
8
Gene Therapy for Spinal Cord Injury . . . . . . . . . . . . . . . . . . . . . . . . . 131 Ioana Goganau and Armin Blesch
9
Gene Therapy for Epilepsy . . . . . . . . . . . . . . .
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