Adeno-associated virus vectors: activity and applications in the CNS

Transgenic strategies are useful for functional studies and they may also lead to novel therapies. Controlling transgene expression in defined cell populations over time is increasingly important for both functional and gene therapy experiments. The adeno-associated virus (AAV) vector may provide sufficient spatio-temporal control of gene expression for these purposes. This paper reviews in vivo somatic gene transfer methodology using AAV. Advantageous features of this system include neuronal gene expression that is: (1) efficient; (2) long-lived; and (3) non-toxic. Thus, AAV-mediated gene transfer is a good method for functional genomic research. From characterizing vector activity in the brain using different combinations of promoters and transgenes in the mid to late 1990s, researchers continue to discover novel uses of AAV for both basic and clinical neuroscience.