CONCEPTS IN GENETIC MEDICINE -- CONTENTS -- CONTRIBUTORS -- PREFACE -- INTRODUCTION -- 1 The Evolution of Human Gene Therapy: A Journey from Excessive Hype to Excessive Diffidence to Reality -- VECTOR SYSTEMS -- 2 Murine Leukemia Virus-Based Retroviral Vectors -- 3 Lentivirus Vectors -- 4 Adenoviral Vectors: History and Perspective -- 5 Adeno-Associated Virus Vectors -- 6 SV40 Virus-Derived Vectors -- 7 Herpes Simplex Virus Vectors -- 8 Nonviral Gene Delivery Systems -- APPLICATIONS -- 9 Therapeutic Gene Transfer to Skeletal Muscle -- 10 Gene Therapy for Cardiovascular Disease -- 11 Intraarticular Vector Delivery for Inflammatory Joint Disease -- 12 The Respiratory System as a Platform for Gene Delivery -- 13 The Brain as a Target for Gene Therapy -- 14 Immune Responses to Viral Vectors Injected Systemically or into the CNS -- 15 Cancer Vaccines -- 16 Genetically Modified T Cells for Human Gene Therapy -- 17 Lentiviral Vector Delivery of RNAi for the Treatment of HIV-1 Infection -- 18 Vector Targeting -- THE MANUFACTURE OF GENE THERAPY PRODUCTS -- 19 The Manufacture of Genetic Viral Vector Products -- 20 The Manufacture of Adeno-Associated Viral Vectors -- 21 Lentivirus Vector Manufacturing -- SAFETY AND REGULATORY CONSIDERATIONS -- 22 Assays for the Release of Viral Vector Gene Therapy Products for Human Clinical Trials -- 23 Safety of Retroviral Vectors: Regulatory and Technical Considerations -- 24 Assays for the Quality Control of Lentiviral Vectors -- 25 Assays for Nonviral Vectors -- 26 Assays for the Release of Cellular Gene Therapy Products -- 27 Toxicology for Gene Therapy Products: Concepts and Challenges -- 28 Regulatory Aspects of Gene Therapy Medicinal Products in the European Union -- FINANCE AND REIMBURSMENT -- 29 Venture Capital and Biotechnology Startups.

30 The Role of Investment Banking in the Growth of Biotechnology Companies -- 31 Managing Reimbursement for Gene Therapy Products -- INDEX.