Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology

This unique advanced textbook provides a clear and comprehensive description of the field of gene delivery, gene therapy and genetic pharmacology, with descriptions of the main gene transfer vectors and a set of selected therapeutic applications, along with safety considerations.The use of gene transfer is exponentially growing in the scientific and medical communities for day-to-day cell biology experiments and swift development of revolutionary gene therapy strategies. In this advanced textbook, more than 25 leading scientists, world-renowned in their respective fields, come together to provide a clear and comprehensive description of gene delivery, gene therapy and genetic pharmacology.This educational introduction to the main gene transfer vectors and selected therapeutic applications provides the background material needed to further explore the subject as well as relevant research literature. It will thus be invaluable to Master, PhD or MD students, post-doctoral scientists or medical doctors, as well as any scientist wishing to deliver a gene or synthetic nucleotide, or develop a gene therapy strategy. Furthermore, the textbook's simple and synthetic content will be of value to any reader interested in the biological and medical revolution derived from the elucidation of the human genome.Sample Chapter(s)Chapter 1: Introduction (52 KB)Contents:Basic Definitions and Principles:Introduction (Thierry VandenDriessche)Basic Definitions and General Principles (Daniel Scherman)History of Gene Therapy (Serge Braun)Genetic Pharmacology Using Synthetic Deoxyribonucleotides (Jean-Christophe François and Carine Giovannangeli)Principles of RNAi Trigger Expression for Gene Therapy (Lisa J Scherer and John J Rossi)On Demand Alternative Splicing for Gene Rescue (Stéphanie Lorain and Luis Garcia)Nuclease-Mediated Targeted Genetic Correction (Dieter C Gruenert, Hamid Emamekhoo and R Geoffrey Sargent)Vectors and Gene Delivery Techniques:γ-Retrovirus- and Lentivirus-Derived Vectors for Gene Transfer and Therapy (Caroline Duros and Odile Cohen-Haguenauer)Adenovirus Vectors (Stefan Kochanek)Adeno-Associated Virus (AAV) Vectors (Aurélie Ploquin, Hildegard Büning and Anna Salvetti)Herpes Simplex Virus (HSV-1)-Based Vectors: Applications for Gene Transfer, Gene Therapy, Cancer Virotherapy and Vaccination (Matias E Melendez, Aldo Pourchet, Anna Greco and Alberto L Epstein)Non-Viral DNA Vectors (Martin Schleef)Macromolecular Conjugates for Non-Viral Nucleic Acid Delivery (Mark Ericson, Kevin Rice and Guy Zuber)Auto-Associative Lipid-Based Systems for Non-Viral Nucleic Acid Delivery (Virginie Escriou, Nathalie Mignet and Andrew Miller)Hydrodynamic-Pressure-Based Non-Viral Nucleic Acid Delivery (Takeshi Suda, Kenya Kamimura, Guisheng Zhang and Dexi Liu)Electrotransfer/Electroporation for Non-Viral Nucleic Acid Delivery (Pascal Bigey, Richard Heller and Daniel Scherman)Imaging of Gene Delivery (Georges Vassaux, Peggy Richard-Fiardo, Béatrice Cambien and Philippe Franken)Therapeutic Applications:Oncolytic Adenoviruses for Cancer Gene Therapy (Gunnel Hallden, Yaohe Wang, Han-Hsi Wong and Nick R Lemoine)Progress in DNA Vaccine Approaches for Cancer Immunotherapy (Geoffrey D Hannigan and David B Weiner)Gene Therapy of the Beta-Hemoglobinopathies (Emmanuel Payen, Charlotte Colomb, Olivier Negre, Marina Cavazzana-Calvo, Salima Hacein-Bey-Abina, Yves Beuzard and Philippe Leboulch)Gene Therapy for Hemophilia A and B (Nisha Nair, Marinee Chuah and Thierry VandenDriessche)Experimental and Clinical Ocular Gene Therapy (Alexis-Pierre Bemelmans and José-Alain Sahel)Gene Therapy of Neurological Diseases (Lisa M Stanek, Lamya S Shihabuddin and Seng H Cheng)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 and George Dickson)Induced Pluripotent Stem...