Abstract
Adeno-associated virus (AAV) continues to be a promising viral vector for delivery of therapeutic genes in human gene therapy. In 2012, the European Commission approved Glybera (alipogene tiparvovec),1 an AAV vector carrying the human lipoprotein lipase gene for commercial use in treatment of lipoprotein lipase deficiency. In addition, within the past five years there have been numerous successes in preclinical, early, and late-stage clinical trials of AAV for treatment of a variety of diseases, including muscular dystrophy, hemophilia, Leber’s congenital amaurosis retinopathy, age-related macular degeneration, cardiac disorders, cancer, and neurodegenerative disorders, such as Parkinson’s disease and Canavan disease. However, despite this progress, the infectious pathway of AAV vectors is an evolving story. In a study recently reported in Nature, Pillay and colleagues2 employed a powerful genome-wide haploid genetic screen to gain a better understanding of the AAV infectious entry pathway. Among other factors consistent with the known pathways of entry and infection, the authors identified a new cellular factor that is required for infection of multiple AAV serotypes, which they term AAV receptor (AAVR).
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