Adeno-associated virus as a gene delivery system.

Adeno-associated virus (AAV) has several characteristics which make it extremely attractive as a gene transfer vector: (1) no known pathogenicity; (2) high efficiency and the ability to remain latent; (3) a minimal number of antigens ensuring minimal immunogenicity; (4) the ability to transduce post-mitotic cells; (5) possible advantages of site-specific integration; and (6) a broad host and cell range. The human isolate, AAV-2, is the best studied and has been the focus for gene delivery experiments. This review will discuss recent in vivo experiments demonstrating the utility of AAV in animal models of neurodegenerative disease.