Concepts in Genetic Medicine

Dr. Boro Dropulic is Founder and Chief Scientific Officer of VIRxSYS, a company that has developed the first lentiviral vector in humans for clinical trial. Dr. Barrie Carter is Senior Vice President and Chief Scientific Officer of Targeted Genetics.

Introduction. Chapter 1: The Evolution of Human Gene Therapy. A Journey from Excessive Hype to Excessive Diffidence to Realty.(Theodore Friedmann). Delivery Systems. Chapter 2: Murine Leukemia Virus Based Vectors.(Kenneth Cornetta). Chapter 3: Lentivirus Vectors (Gwendolyn K. Binder and Boro Dropulic). Chapter 4: Adenoviral Vectors: History and Perspective (Douglas J. Jolly, Estuardo Aguilar-Cordova, and Laura K. Aguilar). Chapter 5: Adeno-associated Virus Vectors (Barrie J. Carter). Chapter 6: The Glass is Half Full: the Goal of Long-term delivery may be Approachable Using SV40-derived Vectors (David S. Strayer). Chapter 7: Herpes Simplex Virus Vectors (Anna-Marie Anesti and Robert S. Coffin). Chapter 8: Nonviral Gene Delivery Systems (Takeshi Suda and Dexi Liu). Applications. Chapter 9: Therapeutic Gene Transfer to Skeletal Muscle (Paul Gregorevic and Jeffrey S. Chamberlain). Chapter 10: Gene Therapy for Cardiovascular Disease (Christina A. Pacak, Kerry O. Cresawn, and Barry J. Byrne). Chapter 11: Intra-articular Vector Delivery for Inflammatory Disease (Haim Burstein). Chapter 12: The Respiratory System as a Platform for Gene Delivery (Barrie J. Carter). Chapter 13: The Brain as a Target for Gene Therapy (Pedro R. Lowenstein and Maria G. Castro). Chapter 14: Immune Responses to Viral vectors Injected Systemically or into the CNS (Pedro R. Lowenstein and Maria G. Castro). Chapter 15: Cancer vaccines (Rimas J. Orentas). Chapter 16: Genetically Modified T cells for Human Gene Therapy (James L. Riley and Carl June). Chapter 17: Lentiviral Vector Delivery of RNAi Triggers for the Treatment of HIV-1 Infection (Kevin V. Morris ands John J. Rossi). Manufacture. Chapter 18: The Manufacture of Genetic Viral Vector Products (Douglas J. Jolly and Estuardo Aguilar-Cordova). Chapter 19: The Manufacture of Adeno-associated Viral Vectors (Richard Peluso). Chapter 20: Lentivirus Vector Manufacturing (Gwendolyn K.Binder and Boro Bropulic). Safety and Regulatory. Chapter 21: Assays for the Release of Viral Vector Gene Therapy Products for Clinical Trials (Flavia Borellini). Chapter 22: Safety of Retroviral Vectors: Regulatory and Technical Considerations (Kenneth Cornetta and Carolyn Wilson). Chapter 23: Assays for the Quality Control of Lentiviral Vectors (James Miskin, Susan M. Kingsman, and Kyriacos A. Mitrophanous). Chapter 24: Assays for Non-viral vectors (Ralph W. Paul). Chapter 25: Assays for the Release of Cellular Gene Therapy Products (Philip J. Cross and Bruce L. Levine). Chapter 26: Toxicology for Gene Therapy Products: Concepts and Challenges (Russette M. Lyons). Chapter 27: Regulatory Aspects of Gene Therapy Medicinal Products in the European Union (Matthias Schweizer, Christian Buchholz and Klaus Cichutek). Finance and Reimbursment. Chapter 28: Venture Capital and Biotechnology Startups (Douglas D. Lind). Chapter 29: The role of Investment Banking in the Growth of Biotechnology Companies (Douglas J. Swirsky). Chapter 30: Managing Reimbursment for Gene Therapy Products (Martin Gold).