Pharmaceutical Aspects of Oligonucleotides PDF
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Pharmaceutical Aspects of Oligonucleotides New and Forthcoming Titles in the Pharmaceutical Sciences Nuclear Medicine in Pharmaceutical Research Perkins and Frier (Eds) 1999 0 7484 0688 3 Hbk Electrically Assisted Transdermal and Topical Drug Delivery Ajay K.Banga 1998 0 7484 0687 5 Hbk Physiological Pharmaceutics Barriers to Drug Absorption 2nd Edition Washington & Wilson 1999 0 7484 0562 3 Hbk 0 7484 0610 7 Pbk Intelligent Software for Product Formulation Rowe & Roberts 1998 0 7484 0732 4 Hbk Flow Injection Analysis of Pharmaceuticals Automation in the Laboratory Martínez Calatayud 1996 0 7484 0445 7 Hbk Photostability of Drugs and Drug Formulations Tønneson (Ed) 1996 0 7484 0449 X Hbk Microbial Quality Assurance in Cosmetics, Toiletries and Non-Sterile Pharmaceuticals 2nd Edition Baird with Bloomfield (Eds) 1996 0 7484 0437 6 Hbk Immunoassay A Practical Guide Law (Ed) 1996 0 7484 0560 7 Hbk Cytochromes P450 Structure, Function and Mechanism Lewis 1996 0 7484 0443 0 Hbk Autonomic Pharmacology Broadley 1996 0 7484 0556 9 Hbk Pharmaceutical Experimental Design and Interpretation 2nd Edition Armstrong & James 1996 0 7484 0436 8 Hbk Pharmaceutical Production Facilities 2nd Edition Cole 1998 0 7484 0438 4 Hbk full pharmaceutical science catalogue available or visit our new website on: http://www.tandf.co.uk 325 Chestnut Street, 8th Floor, Philadelphia, PA 19106, USA Tel: 215–625–8900 Fax: 215–625–2940 11 New Fetter Lane London, EC4P 4EE Tel: +44 (0)20–7583 9855 iii Fax: +44 (0)20–7842 2293 Pharmaceutical Aspects of Oligonucleotides Edited by PATRICK COUVREUR University of Paris-Sud CNRS Unit ‘Physico-chimie, Pharmacotechnie, Biopharmacie’, Chatenay-Malabry, France CLAUDE MALVY CNRS, Institut G.Roussy, Villejuif, France First published 2000 by Taylor & Francis 11 New Fetter Lane, London EC4P 4EE Simultaneously published in the USA and Canada by Taylor & Francis Inc., 325 Chestnut Street, 8th Floor, Philadelphia, PA 19106 Taylor & Francis is an imprint of the Taylor & Francis Group This edition published in the Taylor & Francis e-Library, 2005. “To purchase your own copy of this or any of Taylor & Francis or Routledge’s collection of thousands of eBooks please go to www.eBookstore.tandf.co.uk.” © 2000 Taylor & Francis Limited All rights reserved. No part of this book may be reprinted or reproduced or utilised in any form or by any electronic, mechanical, or other means, now known or hereafter invented, including photocopying and recording, or in any information storage or retrieval system, without permission in writing from the publishers. Every effort has been made to ensure that the advice and information in this book is true and accurate at the time of going to press. However, neither the publisher nor the authors can accept any legal responsibility or liability for any errors or omissions that may be made. In the case of drug administration, any medical procedure or the use of technical equipment mentioned within this book, you are strongly advised to consult the manufacturer’s guidelines. British Library Cataloguing in Publication Data A catalogue record for this book is available from the British Library Library of Congress Cataloging in Publication Data Pharmaceutical aspects of oligonucleotides/edited by Patrick Couvreur, Claude Malvy. p. cm.—(Taylor & Francis series in pharmaceutical sciences) Includes bibliographical references and index. 1. Oligonucleotides—Therapeutic use. I. Couvreur, Patrick. II. Malvy, Claude. III. Series. [DNLM: 1. Oligonucleotides—therapeutic use. 2. Oligonucleotides— adverse effects. QU 57 P536 2000] RM666.N87P47 2000 615′.31—dc21 99–34210 ISBN 0-203-30566-3 Master e-book ISBN ISBN 0-203-34211-9 (Adobe eReader Format) ISBN 0-748-40841-X (Print Edition) Contents Contributors xii Preface xiv PART ONE General Features 1 1 Mechanisms of Action of Antisense 2 Oligonucleotides D.M.Tidd and R.V.Giles 1.1 Introduction 2 1.2 Cell Proliferation Arrest through Release of 3 Deoxynucleosides 1.3 Extracellular Aptameric Effects of Phosphorothioate 5 Oligodeoxynucleotide Analogues 1.4 Aptameric Effects of G-quartet Oligonucleotides and 6 Analogues 1.5 Immune Stimulation by Oligodeoxynucleotides 10 Containing CpG Motifs 1.6 Other Activities of Oligodeoxynucleotides Containing 11 CpG Motifs 1.7 Antisense Inhibition of Gene Expression by Steric 11 Block 1.8 Antisense Inhibition of Gene Expression through 13 Ribonuclease H-mediated Destruction of Target mRNA 1.9 Oligonucleotides in Vivo 20 1.10 Conclusions 21 Acknowledgements 21 References 22 vii PART TWO Chemical Aspects 32 2 Chemistry of Oligonucleotides 33 J.W.Engels and E.Uhlmann 2.1 Introduction 33 2.2 Design of Oligonucleotides 37 2.3 Oligonucleotide Modifications: Synthesis and 40 Properties 2.4 Analysis of Oligonucleotides 59 2.5 Conclusion 68 Acknowledgement 68 References 68 3 The Oligonucleotide Prodrug Approach: The 80 Pro-oligonucleotides F.Morvan, J.-J.Vasseur, E.Vivès, B.Rayner and J.-L.Imbach 3.1 Introduction 80 3.2 What Kind of Enzymolabile Group? 81 3.3 First Pro-oligonucleotide Models 84 3.4 Pro-oligonucleotides of the Second Generation 87 3.5 Conclusion 94 Acknowledgements 96 References 96 4 Peptide Nucleic Acids 100 P.E.Nielsen 4.1 Introduction 100 4.2 Antisense 101 4.3 Antimicrobials 103 4.4 Anti-telomerase 103 4.5 Antigene 103 4.6 Anti-HIV 105 4.7 Pharmacology 105 4.8 Further Developments 106 viii Acknowledgement 106 References 107 PART THREE Delivery 110 5 Peptide-mediated Delivery of Oligonucleotides 111 E.Vivès and B.Lebleu 5.1 Delivery Vehicles for the Improved Uptake of Nucleic 111 Acids: a Survey 5.2 The Potential of Peptides for Nucleic Acids Delivery 113 5.3 Strategies for the Coupling of Peptides to 114 Oligonucleotides 5.4 Poly (L-lysine)-based Delivery Systems 116 5.5 Conjugation to Fusogenic Peptides Allowing 117 Membrane Fusion or Membrane Translocation 5.6 Conclusions 122 Acknowledgements 124 References 124 6 Polymeric Nanoparticles and Microparticles as 129 Carriers for Antisense Oligonucleotides E.Fattal and P.Couvreur 6.1 Introduction 129 6.2 Nanoparticles 130 6.3 In Vitro Stability of ONs Adsorbed onto Nanoparticles 138 6.4 Cell Interactions with ON Loaded Nanoparticles 139 6.5 In Vitro Pharmacological Activity of Oligonucleotide- 140 loaded Nanoparticles 6.6 In Vivo Studies with Oligonucleotide Nanoparticles 140 6.7 Microparticles 141 6.8 Conclusion 142 References 142 7 Liposomes for the Delivery of Oligonucleotides 148 P.Couvreur, C.Malvy, C.Dubernet and E.Fattal 7.1 Introduction 148 ix 7.2 Anionic Liposomes 149 7.3 Cationic Liposomes 152 7.4 pH-sensitive Liposomes 159 7.5 Immunoliposomes and Other Molecularly Targeted 163 Liposomes 7.6 Fusogenic Liposomes and Proteoliposomes 165 7.7 Conclusions 167 References 168 8 Comb-type Polycation Copolymer for Antigene 176 Strategy and DNA Carrier A.Maruyama 8.1 Introduction 176 8.2 Comb-type Polycations as a Stabilizer for DNA 176 Duplex and Triplex 8.3 Comb-type Polycation Copolymers with Cell-specific 189 Polysaccharide Side-chains as cell-specific DNA Carrier Acknowledgements 196 References 196 PART FOUR Biopharmaceutics 203 9 Delivery of Antisense Oligonucleotides in Vitro: 204 Experimental Points G.B.Takle and C.A.Stein 9.1 Introduction 204 9.2 Oligonucleotide-Binding Proteins on the Cell Surface 206 9.3 Intracellular Compartmentalization 208 9.4 Oligonucleotide Delivery Reagents—Practical 210 Considerations 9.5 Experimental Antisense: Points to Consider 211 References 214 10 Mechanisms of Transmembrane Transport of 217 Oligonucleotides
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