Stem Cells in Modeling Human Genetic Diseases
Using stem cells to investigate currently untreatable human genetic diseases is the focus of this book. Several applications of the Nobel-Prize winning, revolutionary iPS cell technology are explored in detail, including in schizophrenia, autism, Huntingt
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Mayana Zatz Keith Okamoto Editors
Stem Cells in Modeling Human Genetic Diseases
Stem Cell Biology and Regenerative Medicine
Series Editor Kursad Turksen, Ph.D. [email protected]
More information about this series at http://www.springer.com/series/7896
Mayana Zatz • Oswaldo Keith Okamoto Editors
Stem Cells in Modeling Human Genetic Diseases
Editors Mayana Zatz Human Genome and Stem-Cell Center (HUG-CELL) Department of Genetics & Evolutionary Biology Institute of Biosciences University of São Paulo São Paulo, Brazil
Oswaldo Keith Okamoto Human Genome and Stem-Cell Center (HUG-CELL) Department of Genetics & Evolutionary Biology Institute of Biosciences University of São Paulo São Paulo, Brazil
ISSN 2196-8985 ISSN 2196-8993 (electronic) Stem Cell Biology and Regenerative Medicine ISBN 978-3-319-18313-8 ISBN 978-3-319-18314-5 (eBook) DOI 10.1007/978-3-319-18314-5 Library of Congress Control Number: 2015943672 Springer Cham Heidelberg New York Dordrecht London © Springer International Publishing Switzerland 2015 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 Humana Press is a brand of Springer Springer Science+Business Media LLC New York is part of Springer Science+Business Media (www.springer.com)
Preface
The cloning of the sheep Dolly in 1996, demonstrating for the first time that it was possible to reprogram a differentiated mammalian cell to a pluripotent state, opened a new and very promising field of research in regenerative medicine. Although the technology of nuclear transfer from a somatic cell to an enucleated egg was banned for human reproductive cloning, it looked very promising for therapeutic cloning and research purposes. However, in practice, it was shown that this technology was extremely challenging and the results were very disappointing and frustrating. The groundbreaking discovery of induced pluripotent stem cells (iPSCs) in mice by Dr. Yamanaka’s group in 2006 came as a surprising alternative to therapeutic clo
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