Adeno-Associated Virus: Methods and Protocols

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, wit...