Adeno-Associated Virus, Softcover reprint of the original 1st ed. 2011 Methods and Protocols Methods in Molecular Biology Series, Vol. 807

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.

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.