Exon Skipping and Inclusion Therapies Methods and Protocols
This book presents a comprehensive collection of detailed state-of-the-art exon skipping and splices modulation protocols. Chapters detail 14 genetic diseases, AON-mediated therapies, and CRISPR/Cas9-mediated gene editing therapies. Written in the highly
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Toshifumi Yokota Rika Maruyama Editors
Exon Skipping and Inclusion Therapies Methods and Protocols
Methods
in
M o l e c u l a r B i o lo g y
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
Exon Skipping and Inclusion Therapies Methods and Protocols
Edited by
Toshifumi Yokota Medical Genetics, University of Alberta, Edmonton, AB, Canada
Rika Maruyama Medical Genetics, University of Alberta, Edmonton, AB, Canada
Editors Toshifumi Yokota Medical Genetics University of Alberta Edmonton, AB, Canada
Rika Maruyama Medical Genetics University of Alberta Edmonton, AB, Canada
ISSN 1064-3745 ISSN 1940-6029 (electronic) Methods in Molecular Biology ISBN 978-1-4939-8650-7 ISBN 978-1-4939-8651-4 (eBook) https://doi.org/10.1007/978-1-4939-8651-4 Library of Congress Control Number: 2018951736 © Springer Science+Business Media, LLC, part of Springer Nature 2018 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. Printed on acid-free paper This Humana Press imprint is published by the registered company Springer Science+Business Media, LLC part of Springer Nature. The registered company address is: 233 Spring Street, New York, NY 10013, U.S.A.
Preface “The man who moves a mountain begins by carrying away small stones.” ―Confucius “The future belongs to those who believe in the beauty of their dreams.” ―Eleanor Roosevelt
Disruption of normal mRNA splicing has been estimated to account for approximately a third of disease-causing mutations. During the past 10 years, antisense-mediated exon skipping/inclusion has proven to be powerful tools for correction of mRNA splicing. In September 2016, the U.S. Food and Drug Administration (FDA) made a historic announcement; they conditionally approved Exondys 51 (eteplirsen), the f
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