Advanced Textbook on Gene Transer, Gene Therapy and Genetic Pharmacology: Principles, Delivery and Pharmacological and Biomedical Applications of Nucleotide-Based Therapies PDF
Preview Advanced Textbook on Gene Transer, Gene Therapy and Genetic Pharmacology: Principles, Delivery and Pharmacological and Biomedical Applications of Nucleotide-Based Therapies
Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology Principles, Delivery and Pharmacological and Biomedical Applications of Nucleotide-Based Therapies ICP Textbooks in Biomolecular Sciences ISSN: 2045-0281 Published Vol. 1: Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology: Principles, Delivery and Pharmacological and Biomedical Applications of Nucleotide-Based Therapies edited by Daniel Scherman (National Scientific Research Center (CNRS), France) Forthcoming title Textbook of Molecular Medicine by Hans Einar Krokan (Norwegian University of Science and Technology, Norway) and Geir Slupphaug (Norwegian University of Science and Technology, Norway) ICP Textbooks in Biomolecular Sciences editor Daniel Scherman National Scientific Research Center (CNRS), France Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology Principles, Delivery and Pharmacological and Biomedical Applications of Nucleotide-Based Therapies Imperial College Press ICP Published by Imperial College Press 57 Shelton Street Covent Garden London WC2H 9HE Distributed by World Scientific Publishing Co. Pte. Ltd. 5 Toh Tuck Link, Singapore 596224 USA office: 27 Warren Street, Suite 401-402, Hackensack, NJ 07601 UK office: 57 Shelton Street, Covent Garden, London WC2H 9HE Library of Congress Cataloging-in-Publication Data Advanced textbook on gene transfer, gene therapy, and genetic pharmacology : principles, delivery, and pharmacological and biomedical applications of nucleotide-based therapies / edited by Daniel Scherman. p. ; cm. -- (ICP textbooks in biomolecular sciences ; v. 1) Includes bibliographical references and index. ISBN 978-1-84816-828-2 (hardcover : alk. paper) ISBN 978-1-908977-28-1 (pbk : alk. paper) I. Scherman, Daniel. II. Series: ICP textbooks in biomolecular sciences ; v. 1. [DNLM: 1. Gene Transfer Techniques. 2. Genetic Therapy--methods. 3. Pharmacogenetics--methods. QZ 52] RB155.8 615.8'95--dc23 2013008317 British Library Cataloguing-in-Publication Data A catalogue record for this book is available from the British Library. Copyright © 2014 by Imperial College Press All rights reserved. This book, or parts thereof, may not be reproduced in any form or by any means, electronic or mechanical, including photocopying, recording or any information storage and retrieval system now known or to be invented, without written permission from the Publisher. For photocopying of material in this volume, please pay a copying fee through the Copyright Clearance Center, Inc., 222 Rosewood Drive, Danvers, MA 01923, USA. In this case permission to photocopy is not required from the publisher. Typeset by Stallion Press Email: [email protected] Printed in Singapore B1574 Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology DEDICATION This textbook is dedicated to my coauthors for their brilliant scientific contributions. I would also like to pay a special tribute to Professors Claude Hélène, Jean-Bernard Le Pecq, and Bernard Roques, members of the French Academy of Sciences, who introduced me to the fields of gene therapy and of pharmacology. My special thanks to my family, especially my wife Sylviane and my children, for sup- porting me through this achievement. bb11557744__FFMM..iinndddd vv 1111//88//22001133 77::3355::0066 AAMM B1574 Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology PREFACE Daniel Scherman, Editor a “In medicinal chemistry, the ruling principle is: Corpora non agunt nisi fi xate, meaning that active substances are only effective if they have a specifi c affi nity for their target” Paul Ehrlich, 1913, in Chemotherapy Medicinal active agents, whose effects are based on the universal concept of “tar- get recognition”. Their history is characterized by a limited number of revolution- ary advances. Except for the traditional medicines derived from the serendipity-driven use of natural plant decoctions, such conceptual revolutions or “quantum leaps” in drug discovery have always resulted from scientific advances. The first revolution resulted from progresses in both analytical and synthetic chemistry, together with the Paul Ehrlich “magic bullet” concept. This has been at the basis of the discovery of chemical drugs directed to a molecularly defined “receptor” target. Indeed, before the 1913 Paul Ehrlich “lock and key” chemo- therapy theory, the concept, today widely accepted, that molecular receptors in living organisms can be selectively affected by chemical compounds was hardly conceivable. Biochemistry breakthroughs and the advances in both protein characterization and recombinant DNA technology paved the way to the use of proteins as thera- peutic agents. Today, recombinant cytokines and hormones, such as erythropoietin (EPO), granulocyte–macrophage colony-stimulating factor (GM-CSF), interferon, and monoclonal antibodies represent a considerable achievement and leading field of drug discovery. a Laboratory of Chemical and Genetic Pharmacology and of Biomedical Imaging, Paris Descartes Pharmacy University, CNRS, Inserm, Chimie ParisTech, 4, avenue de l’Observatoire Paris Cedex 06, France Email: [email protected] vii bb11557744__FFMM..iinndddd vviiii 1111//88//22001133 77::3355::0066 AAMM B1574 Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology viii (cid:132) Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology As an overwhelming rule for both chemical and protein drugs the molecular target in the patient is a protein, with rare exceptions including cytotoxic anti- cancer agents such as cisplatin which bind to DNA independently of the genetic sequence. Representing the most recent revolutionary leap forward, both genetic pharma- cology and gene therapy are based on the use of the genetic code. Genetic pharma- cology represents the critical ultimate step of the Paul Ehrlich “lock and key” concept, in which the drug target is an intracellular genetic sequence within either a DNA or a RNA molecule which is recognized by Watson–Crick or Hogsteen base pairing. By contrast, in gene therapy a gene is administered to the patient’s cells, leading to the transcription by RNA polymerases of a RNA, which can be by itself a therapeutic agent, or most often represents an mRNA translated into a therapeutic protein by the ribosome machinery. In gene therapy, the admin- istered gene can thus be considered as a “prodrug”, with the amplification advan- tage resulting from the continuous intracellular production of the therapeutic RNA and eventually protein. This textbook describes the concepts and applications of genetic pharmacol- ogy and gene therapy. Extensive biological, pharmaceutical and medicinal aspects are presented in a very simple and pedagogical way by world-recognized experts. This rapidly developing field is finely introduced by Professor Thierry VandenDriessche, former President of the European Society of Gene & Cell Therapy. The basic definitions and principles are presented in Part I, while the various vectors and gene-delivery techniques are introduced in Part II. Examples of therapeutic applications are described in Part III, and a more technological Part IV is dedicated to vector production. In addition to chapters written in a pedagogical “textbook style” advising the reader on a few key reviews for further reading, several chapters have been writ- ten as reviews including an extensive bibliography. Therefore, both newcomers and experienced readers might find in the present textbook the most helpful intro- ductory information or more specialized answers to their specific needs. I am honored and grateful that such an assembly of talents has accepted the invitation to contribute to this textbook, which I hope will prove most useful to biological and medicinal students and scientists planning to use gene delivery for basic science or translational medical application, and to medical doctors wishing to be introduced to the exciting new frontline medical fields of genetic pharmacol- ogy and gene therapy. Professor Daniel Scherman bb11557744__FFMM..iinndddd vviiiiii 1111//88//22001133 77::3355::0066 AAMM B1574 Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology CONTENTS Preface vii Part I Basic Definitions and Principles 1 Chapter 1 Introduction 3 Thierry VandenDriessche Chapter 2 Basic Definitions and General Principles 7 Daniel Scherman Chapter 3 History of Gene Therapy 17 Serge Braun Chapter 4 Genetic Pharmacology Using Synthetic Deoxyribonucleotides 31 Jean-Christophe François and Carine Giovannangeli Chapter 5 Principles of RNAi Trigger Expression for Gene Therapy 53 Lisa J. Scherer and John J. Rossi Chapter 6 On Demand Alternative Splicing for Gene Rescue 73 Stéphanie Lorain and Luis Garcia Chapter 7 Nuclease-Mediated Targeted Genetic Correction 85 Dieter C. Gruenert, Hamid Emamekhoo and R. Geoffrey Sargent Part II Vectors and Gene Delivery Techniques 115 γ Chapter 8 -Retrovirus- and Lentivirus-Derived Vectors for Gene Transfer and Therapy 117 Caroline Duros and Odile Cohen-Haguenauer Chapter 9 Adenovirus Vectors 143 Stefan Kochanek ix bb11557744__FFMM..iinndddd iixx 1111//88//22001133 77::3355::0066 AAMM
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