AAV1-CAG-ZsGreen

Adeno-associated virus (AAV) is a single-stranded DNA packaging virus belonging to the Parvoviridae family and is a promising vector for gene delivery. There are currently more than 100 AAV genomic isolates, and 13 human and non-human serotypes have been described. To date, no disease associated with AAV infection has been found. Recombinant AAV (rAAV) vectors can package foreign (non-viral) genes, transduce dividing and non-dividing cells, and induce long-term gene expression in non-dividing cells. In addition, AAV serotypes have different transduction efficiencies for different tissues, depending on their capsid sequences. These properties make AAV an ideal vector for therapeutic gene delivery.

AAV gene delivery systems have been successfully used in several human clinical trials, including the use of rAAV8 vectors to express therapeutic levels of factor IX protein for the treatment of hemophilia B, and the use of rAAV2 vectors encoding a retinal pigment epithelium-specific 65 kDa protein to restore vision in patients with Leber congenital amaurosis. AAV1 has shown better and faster transgene expression in skeletal muscle than AAV2 and has also been used in several clinical trials, including for the treatment of antitrypsin deficiency, lipoprotein lipase deficiency, Pompe disease, and muscular dystrophy. Notably, in 2012, the European Commission approved the use of rAAV1 vectors encoding lipoprotein lipase for the treatment of patients with this enzyme deficiency. This approval represented the first time that gene therapy became a viable clinical treatment.