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Concepts in Genetic Medicine

Boro Dropulic (Editor), Barrie Carter (Editor)

ISBN: 978-0-470-18458-5 April 2007 608 Pages


This book delivers a collection of organized and succinct reviews in the field of therapeutic genetic medicine presented by a carefully selected group of top experts. 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. The book is complemented with uniformly designed illustrations and a supplementary Web site with a PowerPoint presentation for educators.

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).


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).


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).

"The editors have done an excellent job of recruiting prominent authors to contribute. This is a unique publication, with good reviews of each topic." (Doody's Book Reviews, November 2008)