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Non-Viral Vectors for Gene Therapy

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Non-Viral Vectors for Gene Therapy VOLUME EIGHTY NINE ADVANCES IN GENETICS ADVANCES IN GENETICS, VOLUME 89 Serial Editors Theodore Friedmann University of California at San Diego, School of Medicine, USA Jay C. Dunlap The Geisel School of Medicine at Dartmouth, Hanover, NH, USA Stephen F. Goodwin University of Oxford, Oxford, UK VOLUME EIGHTY NINE ADVANCES IN GENETICS Nonviral Vectors for Gene Therapy Physical Methods and Medical Translation Edited by LEAF HUANG Division of Molecular Pharmaceutics and Center for Nanotechnology in Drug Delivery, University of North Carolina at Chapel Hill, Eshelman School of Pharmacy, Chapel Hill, NC, USA DEXI LIU Department of Pharmaceutical and Biomedical Sciences, University of Georgia College of Pharmacy, Athens, GA, USA ERNST WAGNER Munich Center for System-based Drug Research, Center for Nanoscience, Ludwig-Maximilians-Universitat,€ Munich, Germany AMSTERDAM • BOSTON • HEIDELBERG • LONDON NEW YORK • OXFORD • PARIS • SAN DIEGO SAN FRANCISCO • SINGAPORE • SYDNEY • TOKYO Academic Press is an imprint of Elsevier Academic Press is an imprint of Elsevier 225 Wyman Street, Waltham, MA 02451, USA 525 B Street, Suite 1800, San Diego, CA 92101–4495, USA 125 London Wall, London, EC2Y 5AS, UK The Boulevard, Langford Lane, Kidlington, Oxford OX5 1GB, UK First edition 2015 Copyright © 2015 Elsevier Inc. All rights reserved. No part of this publication may be reproduced or transmitted in any form or by any means, electronic or mechanical, including photocopying, recording, or any information storage and retrieval system, without permission in writing from the publisher. Details on how to seek permission, further information about the Publisher’s permissions policies and our arrangements with organizations such as the Copyright Clearance Center and the Copyright Licensing Agency, can be found at our website: www.elsevier.com/permissions. This book and the individual contributions contained in it are protected under copyright by the Publisher (other than as may be noted herein). Notices Knowledge and best practice in this field are constantly changing. As new research and experience broaden our understanding, changes in research methods, professional practices, or medical treatment may become necessary. Practitioners and researchers must always rely on their own experience and knowledge in evaluating and using any information, methods, compounds, or experiments described herein. In using such information or methods they should be mindful of their own safety and the safety of others, including parties for whom they have a professional responsibility. To the fullest extent of the law, neither the Publisher nor the authors, contributors, or editors, assume any liability for any injury and/or damage to persons or property as a matter of products liability, negligence or otherwise, or from any use or operation of any methods, products, instructions, or ideas contained in the material herein. ISBN: 978-0-12-802272-6 ISSN: 0065-2660 For information on all Academic Press publications visit our website at http://store.elsevier.com/ DEDICATION We dedicate this book to Professor Feng Liu, who was murdered on July 24, 2014, for his contribution in establishing the procedure of hydrodynamic gene delivery, the most effective and simplest nonviral method of hepatic gene transfer in vivo developed so far. Huang, Leaf Liu, Dexi Wagner, Ernst This page intentionally left blank CONTENTS Contributors xi 1. Physical Methods for Gene Transfer 1 Mohammad Alsaggar and Dexi Liu 1. Introduction 2 2. Most Commonly Used Physical Methods 4 3. Future Perspectives 15 References 16 2. Nonviral Gene Delivery Systems by the Combination of Bubble Liposomes and Ultrasound 25 Daiki Omata, Yoichi Negishi, Ryo Suzuki, Yusuke Oda, Yoko Endo-Takahashi and Kazuo Maruyama 1. Introduction 26 2. Microbubbles and Ultrasound 27 3. Bubble Liposomes 28 4. In vitro pDNA Delivery with BLs 30 5. In vivo pDNA Delivery with BLs 31 6. In vitro/In vivo Small Interfering RNA Delivery with BLs 33 7. siRNA-Loaded BLs 35 8. MicroRNA-Loaded BLs 38 9. Endosomal Escape Enhanced by BLs 38 10. Conclusions 43 Acknowledgments 43 References 43 3. Electroporation-Mediated Gene Delivery 49 Jennifer L. Young and David A. Dean 1. Introduction 50 2. Theory of Membrane Electroporation 51 3. Mechanism(s) of Electroporation-Mediated Gene Delivery 53 4. Pulse Parameters 58 5. Electrodes 60 6. Tissues that can be Directly Injected: Liver and Skeletal Muscle 63 7. Electroporation of Skeletal Muscle: DNA Vaccines and Use as Bioreactors 65 8. Electroporation of Skin: Vaccination, Wound Healing, and Cancer 67 vii j viii Contents 9. Electroporation of the Cardiovascular System 71 10. Gene Delivery into the Lung Using Electroporation 73 11. Conclusions 78 Acknowledgments 78 References 78 4. Hydrodynamic Delivery 89 Takeshi Suda and Dexi Liu 1. Fundamentals of Hydrodynamic Delivery 89 2. Applications of Hydrodynamic Gene Delivery 95 3. Hydrodynamic Delivery in Large AnimalsdToward Clinic Application 100 4. Future Prospective 103 Acknowledgments 103 References 104 5. Sustained Expression from DNA Vectors 113 Suet Ping Wong, Orestis Argyros and Richard P. Harbottle 1. Introduction 114 2. Immune Responses 115 3. Preventing Epigenetic Silencing 116 4. Position Effect 117 5. Persistent Nonintegrating Episomal DNA Vectors 118 6. Episomal DNA Vectors Based on Replication-Deficient Viruses 118 7. Episomal DNA Vectors Based on Chromosomal Elements 121 8. Mammalian Artificial Chromosomes 121 9. Episomally Maintained pDNA Vectors 122 10. Scaffold/Matrix Attachment Regions 123 11. Insulator Function 124 12. Transcription Augmentation 125 13. Mitotic Stability 127 14. Persistent Expression Mediated by S/MAR DNA Vectors 127 15. Alternative Elements to Modulate Transcription Levels of DNA Vectors 128 16. Optimization of Episomal Vectors for Gene Therapy 129 17. Promoters and Enhancers 130 18. The CMV Promoter 131 19. The UbC Promoter 131 20. The AAT Promoter 132 21. Enhancers 132 22. Posttranscriptional Regulation 133 Contents ix 23. Effect of CpG Depletion 135 24. Studies Demonstrating Passive Episomal State of Minicircles In vivo 138 25. Conclusions 142 References 143 6. Noncoding Oligonucleotides: The Belle of the Ball in Gene Therapy 153 Ka-To Shum and John J. Rossi 1. Introduction 154 2. Noncoding Oligonucleotides-Based Therapeutics in Gene Therapy 155 3. Challenges for Successful Gene Therapy Using Noncoding Oligonucleotides 171 Acknowledgments 172 Conflict of Interest Statement 172 References 172 7. Self-Amplifying mRNA Vaccines 179 Luis A. Brito, Sushma Kommareddy, Domenico Maione, Yasushi Uematsu, Cinzia Giovani, Francesco Berlanda Scorza, Gillis R. Otten, Dong Yu, Christian W. Mandl, Peter W. Mason, Philip R. Dormitzer, Jeffrey B. Ulmer and Andrew J. Geall 1. Introduction 180 2. History of Nucleic Acid Vaccines 181 3. Introduction to mRNA Vaccines 183 4. Self-Amplifying mRNA 184 5. Delivery of Self-Amplifying mRNA Vaccines 185 6. Production and Purification of Self-Amplifying mRNA 211 7. Stability of mRNA 215 8. Future Prospects for Nonviral Delivery of Self-Amplifying mRNA Vaccines 220 Acknowledgments 222 References 222 8. Gene Electrotransfer Clinical Trials 235 Richard Heller and Loree C. Heller 1. Introduction 236 2. GET in Clinical Trials 237 3. Summary and Conclusions 254 References 257 Index 263 This page intentionally left blank CONTRIBUTORS Mohammad Alsaggar Department of Pharmaceutical and Biomedical Sciences, University of Georgia College of Pharmacy, Athens, GA, USA Orestis Argyros Division of Pharmacology–Pharmacotechnology, Biomedical Research Foundation of the Academy of Athens, Athens, Greece Francesco Berlanda Scorza Novartis Vaccines, Inc., Holly Springs, NC, USA Luis A. Brito Novartis Vaccines, Inc., Cambridge, MA, USA David A. Dean Departments of Pediatrics and Biomedical Engineering, University of Rochester, Rochester, NY, USA Philip R. Dormitzer Novartis Vaccines, Inc., Cambridge, MA, USA Yoko Endo-Takahashi Department of Drug Delivery and Molecular Biopharmaceutics, School of Pharmacy, Tokyo University of Pharmacy and Life Sciences, Hachioji, Tokyo, Japan Andrew J. Geall Novartis Vaccines, Inc., Cambridge, MA, USA Cinzia Giovani Novartis Vaccines, Inc., Siena, Italy Richard P. Harbottle DNA Vector Research, German Cancer Research Centre (DKFZ), Heidelberg, Germany Loree C. Heller Frank Reidy Research Center for Bioelectrics, Old Dominion University, School of Medical Diagnostics and Translational Sciences, College of Health Sciences, Norfolk, VA, USA Richard Heller Frank Reidy Research Center for Bioelectrics, Old Dominion University, School of Medical Diagnostics and Translational Sciences, College of Health Sciences, Norfolk, VA, USA Sushma Kommareddy Novartis Vaccines, Inc., Cambridge, MA, USA xij xii Contributors Dexi Liu Department of Pharmaceutical and Biomedical Sciences, University of Georgia College of Pharmacy, School of Pharmacy, Athens, GA, USA Domenico Maione Novartis Vaccines, Inc., Siena, Italy Christian W. Mandl Novartis Vaccines, Inc., Cambridge, MA, USA Kazuo Maruyama Department of Drug and Gene Delivery Research, Faculty of Pharma-Sciences, Teikyo University, Itabashi, Tokyo, Japan Peter W. Mason Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA Yoichi Negishi Department of Drug Delivery and Molecular Biopharmaceutics, School of Pharmacy, Tokyo University of Pharmacy and Life Sciences, Hachioji, Tokyo, Japan Yusuke Oda Department of Drug and Gene Delivery Research, Faculty of Pharma-Sciences, Teikyo University, Itabashi, Tokyo, Japan Daiki Omata Department of Drug and Gene Delivery
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