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Full Description
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™ 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.
Authoritative and accessible, Adeno-Associated Virus: Methods and Protocols provides a complete and comprehensive understanding of this multi-disciplinary and rapidly progressing field.
Contents
Adeno-Associated Virus Biology.- Design and Construction of Functional AAV Vectors.- AAV Capsid Structure and Cell Interactions.- Exploiting Natural Diversity of AAV for the Design of Vectors With Novel Properties.- Gene Therapy in Skeletal Muscle Mediated by Adeno-Associated Virus (AAV) Vectors.- AAV-Mediated Liver-Directed Gene Therapy.- Recombinant AAV Delivery to the Central Nervous System.- AAV Mediated Gene Therapy for Retinal Degenerative Diseases.- Adeno-Associated Virus Vector Delivery to the Heart.- Evaluation of the Fate of rAAV Genomes Following in vivo Administration.- Measuring Immune Responses to Recombinant AAV Gene Transfer.- Modification and Labeling of AAV Vector Particles.- AAV-mediated Gene Targeting.- Preclinical Study Design for rAAV.- Biodistribution and Shedding of AAV Vectors.- Production and Purification of Recombinant Adeno-Associated Vectors.- rAAV Vector Product Characterization and Stability Studies.- rAAV Human Trial Experience.
