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Adeno-Associated Virus: Methods and Protocols

Adeno-Associated Virus: Methods and Protocols

Hardcover

Series: Methods in Molecular Biology, Book 807

Medical ReferenceGenetics

ISBN10: 1617793698
ISBN13: 9781617793691
Publisher: Humana
Published: Oct 28 2011
Pages: 462
Weight: 2.15
Height: 1.10 Width: 7.00 Depth: 10.10
Language: English

Today, progress in rAAV-mediated gene transfer is so robust that long-term, efficient, and regulatable transgene expression is reproducibly achieved in large animal models. The complexity of gene transfer agents in the context of their clinical use requires investigators from a wide variety of backgrounds to have an understanding - or at least an appreciation of - the regulatory environment and constraints that affect vector design, manufacturing, pre-clinical testing, and clinical use, with an emphasis on patient protection. In Adeno-Associated Virus: Methods and Protocols, experts from the United States and Europe have contributed current knowledge of this multi-dimensional field relating to the biology of AAV, rAAV vector design, vector manufacturing and product testing, performance of rAAV vectors in major organs, rAAV-related immunological issues, design of animal and clinical studies, and clinical experience. Written in the successful Methods in Molecular Biology(TM) series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible protocols, and notes on troubleshooting and avoiding known pitfalls.

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