Syn-NES-jRCaMP1a AAV (Serotype 9)

Adeno-associated virus (AAV) is a non-pathogenic parvovirus that is widely used as a viral vector for gene therapy due to its safety and simplicity. Recombinant AAV vectors are one of the most popular vectors for therapeutic gene delivery and have been developed for many years. To date, 13 AAV serotypes and more than 100 variants have been identified. The AAV genome can persist in the nucleus of transduced cells and induce stable transgene expression in different tissues and species. Therefore, AAV-mediated gene delivery has been widely studied preclinically. Several AAV serotypes or variants, including AAV1, 2, 4, 5, 8, 9, and rh10, have been used in clinical trials.

Previous studies have shown that different tissue biodistributions of AAV serotypes depend on the delivery route. Several serotypes of AAV vectors are able to effectively induce global transduction with a single intravascular injection. Compared with other serotypes, AAV9 is superior in viral genome distribution and protein expression after systemic administration. AAV9 is able to effectively transduce to the brain, spinal cord, muscle, and other tissues after peripheral intravenous injection. Therefore, systemic administration of AAV9 vectors has been proposed and applied in clinical trials for patients with central nervous system disorders and muscle diseases, including amyotrophic lateral sclerosis, frontotemporal dementia, Rett syndrome, Huntington's disease, and muscular dystrophy.