Genome Editing
Kursad Turksen Editor Genome Editing
Editor Kursad Turksen Ottawa, ON, Canada ISBN 978-3-319-34146-0 ISBN 978-3-319-34148-4 (ebook) DOI 10.1007/978-3-319-34148-4 Library of Congress Control Number: 2016945569 Springer International Publishing Switzerland 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 This Springer imprint is published by Springer Nature The registered company is Springer International Publishing AG Switzerland
Contents Genome Editing with Targetable Nucleases... 1 Stephane Pelletier CRISPR/Cas9 Approaches to Investigate the Noncoding Genome... 31 Davide Seruggia and Lluis Montoliu At the Conflux of Human Genome Engineering and Induced Pluripotency... 45 Knut Woltjen, Fabian Oceguera-Yanez, Harunobu Kagawa, and Shin-Il Kim CRISPR/Cas9 and the Paradigm Shift in Mouse Genome Manipulation Technologies... 65 Channabasavaiah B. Gurumurthy, Rolen M. Quadros, Masahiro Sato, Tomoji Mashimo, K.C. Kent Lloyd, and Masato Ohtsuka Genome-Editing Technology in CRISPR/Cas System: How to Increase Knock-In Efficiency in Mouse Zygotes... 79 Takuro Horii and Izuho Hatada Developments in the Generation of Reporter Stem Cells... 93 Samvel Avagyan, Kyle Sylakowski, and Uma Lakshmipathy Current Status of Genome Editing in Cardiovascular Medicine... 107 Vittavat Termglinchan, Ioannis Karakikes, Timon Seeger, and Joseph C. Wu Using CRISPR-Cas9 Genome Editing to Enhance Cell Based Therapies for the Treatment of Diabetes Mellitus... 127 Nicole A.J. Krentz and Francis C. Lynn Genome Editing in the Retina: A Case Study in CRISPR for a Patient-Specific Autosomal Dominant Retinitis Pigmentosa Model... 149 Sally Justus, Andrew Zheng, Yi-Ting Tsai, Wen-Hsuan Wu, Chun-Wei Hsu, Wei-Pu Wu, Alexander G. Bassuk, Vinit B. Mahajan, and Stephen H. Tsang Index...... 163 v
Contributors Samvel Avagyan Life Sciences Solutions, Thermo Fisher Scientific, Carlsbad, CA, USA CIRM Bridges Program, San Diego State University, San Diego, CA, USA Rensselaer Polytechnic Institute, Troy, NY, USA Alexander G. Bassuk Department of Pediatrics, The University of Iowa Children s Hospital, Iowa City, IA, USA Channabasavaiah B. Gurumurthy Developmental Neuroscience, Munroe Meyer Institute for Genetics and Rehabilitation, University of Nebraska Medical Center, Omaha, NE, USA Mouse Genome Engineering Core Facility, Office of the Vice Chancellor for Re-search, University of Nebraska Medical Center, Omaha, NE, USA Izuho Hatada Laboratory of Genome Science, Biosignal Genome Resource Center, Institute for Molecular and Cellular Regulation, Gunma University, Maebashi, Gunma, Japan Takuro Horii Laboratory of Genome Science, Biosignal Genome Resource Center, Institute for Molecular and Cellular Regulation, Gunma University, Maebashi, Gunma, Japan Chun-Wei Hsu Department of Ophthalmology, College of Physicians and Surgeons, Sally Justus Department of Ophthalmology, College of Physicians and Surgeons, Harunobu Kagawa Center for ips Cell Research and Application (CiRA), Kyoto University, Kyoto, Japan vii
viii Contributors Ioannis Karakikes Stanford Cardiovascular Institute, Stanford University School of Division of Cardiology, Department of Medicine, Stanford University School of Shin-Il Kim Center for ips Cell Research and Application (CiRA), Kyoto University, Kyoto, Japan Nicole A.J. Krentz Diabetes Research Group, Child and Family Research Institute, Vancouver, BC, Canada Departments of Surgery/Cellular and Physiological Sciences, University of British Columbia, Vancouver, BC, Canada Uma Lakshmipathy Life Sciences Solutions, Thermo Fisher Scientific, Carlsbad, CA, USA K.C. Kent Lloyd Mouse Biology Program, University of California, Davis, Davis, CA, USA Francis C. Lynn Diabetes Research Group, Child and Family Research Institute, Vancouver, BC, Canada Departments of Surgery/Cellular and Physiological Sciences, University of British Columbia, Vancouver, BC, Canada Vinit B. Mahajan Department of Ophthalmology, Iowa University Hospitals and Clinics, Iowa City, IA, USA Tomoji Mashimo Institute of Experimental Animal Sciences, Graduate School of Medicine, Osaka University, Osaka, Japan Lluis Montoliu Department of Molecular and Cellular Biology, National Centre for Biotechnology (CNB-CSIC), Madrid, Spain CIBERER-ISCIII, Madrid, Spain Fabian Oceguera-Yanez Center for ips Cell Research and Application (CiRA), Kyoto University, Kyoto, Japan Masato Ohtsuka Department of Molecular Life Science, Division of Basic Medical Science and Molecular Medicine, Tokai University School of Medicine, Isehara, Kanagawa, Japan Stephane Pelletier Department of Immunology, St. Jude Children s Research Hospital, Memphis, TN, USA Rolen M. Quadros Mouse Genome Engineering Core Facility, Office of the Vice Chancellor for Research, University of Nebraska Medical Center, Omaha, NE, USA Masahiro Sato Section of Gene Expression Regulation, Frontier Science Research Center, Kagoshima University, Kagoshima, Japan
Contributors ix Timon Seeger Stanford Cardiovascular Institute, Stanford University School of Division of Cardiology, Department of Medicine, Stanford University School of Davide Seruggia Department of Molecular and Cellular Biology, National Centre for Biotechnology (CNB-CSIC), Madrid, Spain CIBERER-ISCIII, Madrid, Spain Department of Hematology/Oncology, Boston Children s Hospital, Boston, MA, USA Kyle Sylakowski Life Sciences Solutions, Thermo Fisher Scientific, Carlsbad, CA, USA University of Pittsburgh, Pittsburgh, PA, USA Vittavat Termglinchan Stanford Cardiovascular Institute, Stanford University School of Division of Cardiology, Department of Medicine, Stanford University School of Yi-Ting Tsai Department of Ophthalmology, College of Physicians and Surgeons, Stephen H. Tsang Department of Ophthalmology, College of Physicians and Surgeons, Department of Pathology and Cell Biology, College of Physicians and Surgeons, Edward S. Harkness Eye Institute, New York-Presbyterian Hospital/Columbia University Medical Center, New York, NY, USA Knut Woltjen Center for ips Cell Research and Application (CiRA), Kyoto University, Kyoto, Japan Hakubi Center for Advanced Research, Kyoto University, Kyoto, Japan Wei-Pu Wu Department of Ophthalmology, College of Physicians and Surgeons, Wen-Hsuan Wu Department of Ophthalmology, College of Physicians and Surgeons, Joseph C. Wu Stanford Cardiovascular Institute, Stanford University School of Division of Cardiology, Department of Medicine, Stanford University School of Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Andrew Zheng Department of Ophthalmology, College of Physicians and Surgeons,