Muscle Gene Therapy
About 7 million people worldwide are suffering from various inherited neuromuscular diseases. Gene therapy brings the hope of treating these diseases at their genetic roots. Muscle Gene Therapy is the only book dedicated to this topic. The fir
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Muscle Gene Therapy Second Edition
Muscle Gene Therapy
Dongsheng Duan • Jerry R. Mendell Editors
Muscle Gene Therapy Second Edition
Editors Dongsheng Duan Department of Molecular Microbiology and Immunology School of Medicine University of Missouri Columbia, MO, USA Department of Neurology School of Medicine University of Missouri Columbia, MO, USA Department of Biomedical Sciences College of Veterinary Medicine University of Missouri Columbia, MO, USA
Jerry R. Mendell Department of Pediatrics
Nationwide Children’s Hospital and Research Institute The Ohio State University Columbus, OH, USA Department of Neurology Nationwide Children’s Hospital and Research Institute The Ohio State University Columbus, OH, USA
Department of Bioengineering University of Missouri Columbia, MO, USA
ISBN 978-3-030-03094-0 ISBN 978-3-030-03095-7 (eBook) https://doi.org/10.1007/978-3-030-03095-7 Library of Congress Control Number: 2019931907 © Springer Nature Switzerland AG 2010, 2019 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. The publisher remains neutral with regard to jurisdictional claims in published maps and institutional affiliations. Cover illustration: Top panels (from left to right): (1) AAV1 gene therapy restored α-sarcoglycan expression in a type 2D limb girdle muscular dystrophy patient; (2) Nominal α-sarcoglycan was detected in patient’s muscle before AAV1 gene therapy; (3) AAV9 micro-dystrophin gene therapy improved muscle histology in the dog model of Duchenne muscular dystrophy; (4) Untreated dystrophic dog muscle showed degeneration, necrosis, inflammation and fibrosis. Bottom panel background: Transmission electron microscope image of purified recombinant adeno-associated virus (AAV) particles. This Springer imprint is published by the registered company Springer Nature Switzerland AG The registered company address is: Gewerbestrasse 11, 6330 Cham, Switzerland
To patients and their families and friends who have fought fiercely to defeat muscle diseases To
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