Muscle Gene Therapy
Muscle disease represents an important health threat to the general population. For thousands of years, a cure has been deemed extremely remote, if not impossible, for many relentless muscle diseases such as Duchenne muscular dystrophy. The cloning of mus
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Dongsheng Duan Editor
Muscle Gene Therapy
Editor Dongsheng Duan University of Missouri Columbia, MO USA [email protected]
ISBN 978-1-4419-1205-3 e-ISBN 978-1-4419-1207-7 DOI 10.1007/978-1-4419-1207-7 Springer New York Dordrecht Heidelberg London Library of Congress Control Number: 2009933117 © Springer Science+Business Media, LLC 2010 All rights reserved. This work may not be translated or copied in whole or in part without the written permission of the publisher (Springer Science+Business Media, LLC, 233 Spring Street, New York, NY 10013, USA), except for brief excerpts in connection with reviews or scholarly analysis. Use in connection with any form of information storage and retrieval, electronic adaptation, computer software, or by similar or dissimilar methodology now known or hereafter developed is forbidden. The use in this publication of trade names, trademarks, service marks, and similar terms, even if they are not identified as such, is not to be taken as an expression of opinion as to whether or not they are subject to proprietary rights. Cover image: Background, transmission electron microscope image of purified recombinant adeno-associated virus (AAV) serotype-5 particles. Right top panel, an AAV serotype-9 virus carrying an alkaline phosphatase gene expression cassette was injected to the jugular vein of a newborn dog. Alkaline phosphatase expression was examined in skeletal muscle six months later on cryo-tissue section by histochemical staining. Right bottom panel, an AAV serotype-6 virus carrying a LacZ gene expression cassette was directly injected to the anterior tibialis muscle in a 2-month-old C57Bl/10 mouse. LacZ expression was examined one month later on cryo-tissue section by b-galactosidase staining. Left panels, an AAV serotype-6 virus carrying a micro-dystrophin gene expression cassette was directly injected to the extensor digitorium longus muscle in a 1-month-old utrophin/dystrophin double knockout mouse, a mouse model for Duchenne muscular dystrophy. Muscle histopathology and micro-dystrophin expression were examined at 1 month after gene therapy. Top panel, hematoxylin-eosin staining. Bottom panel, immunofluorescence staining for micro-dystrophin expression. Printed on acid-free paper Springer is part of Springer Science+Business Media (www.springer.com)
To patients, their families and friends who have fought fiercely to defeat muscle diseases To investigators who work diligently to find the cure for muscle diseases
Preface
Scene 1. The year was 1890. A young couple brought their 5-year-old son to their physician. Unlike his peers, the boy had a hard time walking and his muscles were getting weaker. “What is the problem? Can you treat it?” the couple implored the physician. In the face of these serious questions from the apprehensive couple, the physician told them he had very little to offer. All he could tell them was that this was a muscle wasting disease commonly seen in young boys. A French physician named Duchenne de Boulogne described this disease in 1868 and it has
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