Translational Research in Muscular Dystrophy
This book presents recent advances in translational research on muscular dystrophy (MD) to physicians and researchers, including cutting-edge research on the disease such as regenerative medicine, next-generation DNA sequencing, and nucleic acid therapies
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Shin’ichi Takeda Yuko Miyagoe-Suzuki Madoka Mori-Yoshimura Editors
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Translational Research in Muscular Dystrophy
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Shin’ichi Takeda • Yuko Miyagoe-Suzuki • Madoka Mori-Yoshimura Editors
Translational Research in Muscular Dystrophy
Editors Shin’ichi Takeda National Institute of Neuroscience Kodaira, Tokyo Japan
Yuko Miyagoe-Suzuki National Institute of Neuroscience Kodaira, Tokyo Japan
Madoka Mori-Yoshimura National Institute of Neuroscience Kodaira, Tokyo Japan
ISBN 978-4-431-55677-0 ISBN 978-4-431-55678-7 DOI 10.1007/978-4-431-55678-7
(eBook)
Library of Congress Control Number: 2015957591 Springer Tokyo Heidelberg New York Dordrecht London © Springer Japan 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 Japan KK is part of Springer Science+Business Media (www.springer.com)
Preface
Since identification of the DMD (dystrophin) gene as the cause of Duchenne muscular dystrophy (DMD) in 1987, many genes have been found to contribute to muscular dystrophies. In particular, recent next-generation sequencing technology has accelerated the identification of disease-causing genes, especially for α-dystroglycanopathies. In parallel, new research tools for revealing the molecular and cellular mechanisms of muscular dystrophies have been extensively investigated, and new therapeutic strategies for treatment of muscular dystrophy, such as exon skipping therapy using antisense-oligonucleotides or stem cell-based therapy using pluripotent stem cells, have been proposed. To support development of therapies and nationwide and international clinical studies for rare, intractable neuromuscular diseases, the EU started the TREAT-NMD network and the United States started a cooperative international neuromuscular research group (CINRG). In Japan, a patient registry site (registry of muscular dystrophy: REMUDY) (http:// www.remudy.jp/) and a muscular dystrophy clinical trial network (http://www. mdctn.jp/network.ht
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