Table of Contents
Preface vii
Part I Basic Definitions and Principles 1
Chapter 1 Introduction Thierry VandenDriessche 3
Chapter 2 Basic Definitions and General Principles Daniel Scherman 7
Chapter 3 History of Gene Therapy Serge Braun 17
Chapter 4 Genetic Pharmacology Using Synthetic Deoxyribonucleotides Jean-Christophe François Carine Giovannangeli 31
Chapter 5 Principles of RNAi Trigger Expression for Gene Therapy Lisa J. Scherer John J. Rossi 53
Chapter 6 On Demand Alternative Splicing for Gene Rescue Stéphanie Lorain Luis Garcia 73
Chapter 7 Nuclease-Mediated Targeted Genetic Correction Dieter C. Grueneri Hamid Emamekhoo R. Geoffrey Sargent 85
Part II Vectors and Gene Delivery Techniques 115
Chapter 8 γ-Retrovirus- and Lentivirus-Derived Vectors for Gene Transfer and Therapy Caroline Duros Odile Cohen-Haguenauer 117
Chapter 9 Adenovirus Vectors Stefan Kochanek 143
Chapter 10 Adeno-Associated Virus (AAV) Vectors Aurélie Ploquin Hildegard Büning Anna Salvetti 151
Chapter 11 Herpes Simplex Virus (HSV-1)-Based Vectors: Applications for Gene Transfer, Gene Therapy, Cancer Virotherapy and Vaccination Matias E. Melendez Aldo Pourchet Anna Greco Alberto L. Epstein 165
Chapter 12 Non-Viral DNA Vectors Martin Schleef 183
Chapter 13 Macromolecular Conjugates for Non-Viral Nucleic Acid Delivery Mark Ericson Kevin Rice Guy Zuber 207
Chapter 14 Auto-Associative Lipid-Based Systems for Non-Viral Nucleic Acid Delivery Virginie Escriou Nathalie Mignet Andrew Miller 221
Chapter 15 Hydrodynamic-Pressure-Based Non-Viral Nucleic Acid Delivery Takeshi Suda Kenya Kamimura Guisheng Zhang Dexi Liu 255
Chapter 16 Electrotransfer/Electroporation for Non-Viral Nucleic Acid Delivery Pascal Bigey Richard Heller Daniel Scherman 265
Chapter 17 Imaging of Gene Delivery Georges Vassaux Peggy Richard-Fiardo Béatrice Cambien Philippe Franken 287
Part III Therapeutic Applications 305
Chapter 18 Oncolytic Adenoviruses for Cancer Gene Therapy Gunnel Hallden Yaohe Wang Han-Hsi Wong Nick R. Lemoine 307
Chapter 19 Progress in DNA Vaccine Approaches for Cancer Immunotherapy Geoffrey D. Hannigan David B. Weiner 331
Chapter 20 Gene Therapy of the Beta-Hemoglobinopathies Emmanuel Payen Charlotte Colomb Olivier Negre Marina Cavazzana-Calvo Salima Hacein-Bey-Abina Yves Beuzard Philippe Leboulch 361
Chapter 21 Gene Therapy for Hemophilia A and B Nisha Nair Marinec Chuah Thierry VandenDriessche 391
Chapter 22 Experimental and Clinical Ocular Gene Therapy Alexis-Pierre Bemelmans José-Alain Sahel 403
Chapter 23 Gene Therapy of Neurological Diseases Lisa M. Stanek Lamya S. Shihabuddin Seng H. Cheng 421
Chapter 24 Genetic Therapy of Muscle Diseases: Duchenne Muscular Dystrophy Takis Athanasopoulos Susan Jarmin Helen Foster Keith Foster Jagjeet Kang Taeyoung Koo Alberto Malerba Linda Popplewell Daniel Scherman George Dickson 453
Chapter 25 Induced Pluripotent Stem Cells and Gene Targeting for Regenerative Medicine Jizhong Zou Linzhao Cheng 477
Part IV Gene Vector Production 491
Chapter 26 Production and Purification of Viral Vectors and Safety Considerations Related to Their Use Otto-Wilhelm Merten Matthias Schweizer Parminder Chahal Amine Kamen 493
Chapter 27 Production and Purification of Plasmid Vectors Martin Schleef 517
Index 527
