Concepts in Genetic Medicine highlights the most important issues in the development, manufacture, and commercialization of novel genetic medicines.

Each chapter focuses on a single, current topic and explains issues in the development of genetic treatments, critical challenges and strategies for implementing them, and future directions in research and translational applications.

Beginning with an introduction to the genetic basis of disease and a discussion of specific diseases, their etiology, and promising therapeutic treatments, the book goes on to cover vector design, genetic payloads and disease targets, vaccine vector systems, manufacture, assays, cell systems and target issues, preclinical testing, regulatory considerations, clinical evaluation, safety concerns, and commercialization.

Contents

1. The Evolution of Human Gene Therapy. A Journey from Excessive Hype to Excessive Diffidence to Realty

Delivery Systems

2. Murine Leukemia Virus Based Vectors
3. Lentivirus Vectors
4. Adenoviral Vectors: History and Perspective
5. Adeno-associated Virus Vectors
6. The Glass is Half Full: the Goal of Long-term delivery may be Approachable Using SV40-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. Intra-articular Vector Delivery for Inflammatory 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 Triggers for the Treatment of HIV-1 Infection

Manufacture

18. The Manufacture of Genetic Viral Vector Products
19. The Manufacture of Adeno-associated Viral Vectors
20. Lentivirus Vector Manufacturing

Safety and Regulatory

21. Assays for the Release of Viral Vector Gene Therapy Products for Clinical Trials
22. Safety of Retroviral Vectors: Regulatory and Technical Considerations
23. Assays for the Quality Control of Lentiviral Vectors
24. Assays for Non-viral vectors
25. Assays for the Release of Cellular Gene Therapy Products
26. Toxicology for Gene Therapy Products: Concepts and Challenges
27. Regulatory Aspects of Gene Therapy Medicinal Products in the European Union

Finance and Reimbursment

28. Venture Capital and Biotechnology Startups
29. The role of Investment Banking in the Growth of Biotechnology Companies
30. Managing Reimbursment for Gene Therapy Products

Index

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