Stem Cell Therapy in Lysosomal Storage Diseases

Stem Cell therapy for lysosomal diseases (LSDs) is developing rapidly. This volume discusses the history, current practice and future perspectives of stem cells in inborn errors of metabolism (IEM) and provides an international perspective on progress, li

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Jaap Jan Boelens Robert Wynn Editors

Stem Cell Therapy in Lysosomal Storage Diseases

Stem Cell Biology and Regenerative Medicine

Series Editor Kursad Turksen, Ph.D. [email protected]

For further volumes: http://www.springer.com/series/7896

Jaap Jan Boelens



Robert Wynn

Editors

Stem Cell Therapy in Lysosomal Storage Diseases

Editors Jaap Jan Boelens Wilhelmina Childrens Hospital University Medical Center Utrecht

Robert Wynn Royal Manchester Children’s Hospital

Manchester, UK

Utrecht, The Netherlands

ISSN 2196-8985 ISSN 2196-8993 (electronic) ISBN 978-1-4614-8356-4 ISBN 978-1-4614-8357-1 (eBook) DOI 10.1007/978-1-4614-8357-1 Springer New York Heidelberg Dordrecht London Library of Congress Control Number: 2013949150 © Springer Science+Business Media New York 2013 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. Exempted from this legal reservation are brief excerpts in connection with reviews or scholarly analysis or material supplied specifically for the purpose of being entered and executed on a computer system, for exclusive use by the purchaser of the work. Duplication of this publication or parts thereof is permitted only under the provisions of the Copyright Law of the Publisher’s location, in its current version, and permission for use must always be obtained from Springer. Permissions for use may be obtained through RightsLink at the Copyright Clearance Center. Violations are liable to prosecution under the respective Copyright Law. 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. While the advice and information in this book are believed to be true and accurate at the date of publication, neither the authors nor the editors nor the publisher can accept any legal responsibility for any errors or omissions that may be made. The publisher makes no warranty, express or implied, with respect to the material contained herein. Printed on acid-free paper Humana Press is a brand of Springer Springer is part of Springer Science+Business Media (www.springer.com)

During the execution of this book the world has lost a leader in the field of Lysosomal Storage Disorders. Ed Wraith, one of the key contributors to the book - died suddenly in Manchester in April 2013. Ed was a pioneer in every aspect of LSDs including cellular therapy. Indeed Jaap Jan Boelens and Rob Wynn first met each other at a meeting attended and chaired by Ed to facilitate collaboration between Europ