title: Adeno-associated virus at 50: a golden anniversary of discovery, research, and gene therapy success--a personal perspective.
abstract: Fifty years after the discovery of adeno-associated virus (AAV) and more than 30 years after the first gene transfer experiment was conducted, dozens of gene therapy clinical trials are in progress, one vector is approved for use in Europe, and breakthroughs in virus modification and disease modeling are paving the way for a revolution in the treatment of rare diseases, cancer, as well as HIV. This review will provide a historical perspective on the progression of AAV for gene therapy from discovery to the clinic, focusing on contributions from the Samulski lab regarding basic science and cloning of AAV, optimized large-scale production of vectors, preclinical large animal studies and safety data, vector modifications for improved efficacy, and successful clinical applications.
authors: 1Gene Therapy Center, University of North Carolina, Chapel Hill, NC 27599-7352.
authors: Eric
authors: E
authors: Hastie
authors: 1Gene Therapy Center, University of North Carolina, Chapel Hill, NC 27599-7352.|2Department of Pharmacology, University of North Carolina, Chapel Hill, NC 27599-7352.
authors: R Jude
authors: RJ
authors: Samulski
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D000229:Dependovirus / Q000235:genetics*; D004353:Drug Evaluation, Preclinical; D018014:Gene Transfer Techniques / Q000266:history; D015316:Genetic Therapy / Q000266:history / Q000379:methods; D005822:Genetic Vectors / Q000235:genetics* / Q000266:history; D049673:History, 20th Century; D049674:History, 21st Century; D006801:Humans; D012106:Research / Q000266:history; D057170:Translational Research, Biomedical