A Novel Adeno-Associated Virus for Gene Therapy

Scientists at the NIH disclosed a novel adeno-associated virus (AAV) termed "44-9." AAV44-9 based vectors have high gene transfer activity in a number of cell types, including salivary gland cells, liver cells, and different types of neurons (e.g., cells of the cortex, olfactory bulb, and brain stem, and Purkinje cells of the cerebellum). These vectors can increase the transduction efficiency and decrease the potential of being neutralized by preexisting antibodies compared to the wild type AAV. Preliminary results from animal studies suggest that AAV44-9 vectors can efficiently deliver genes of interest, and the protein products of the delivered genes can be detected in bloodstream and at the local tissues. Therefore, these vectors are suitable for gene therapy for cells/tissues that are not efficiently targeted by other vectors.