Concepts in Genetic Medicine

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Contenidos del Libro:

Part I: Introduction

1.- The Evolution of Human Gene Therapy: A Journey from Excessive Hype to Excessive Diffidence to Reality

Part II: 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

Part III: 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

Part IV: 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

Part V: Safety and Regulatory Considartions
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

Part VI: 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

 Actualización: 24/09/2012