AAV6-CAG-FLPo

Adeno-associated virus (AAV) is a small (25 nm), non-replicating, non-pathogenic, non-enveloped parvovirus that attaches to cells primarily via heparin sulfate proteoglycan receptors and is then internalized by endocytosis. After endocytosis, AAV is transported to the nucleus, where it is uncoated and releases the vector genome. Once in the host cell nucleus, the AAV vector genome forms persistent extrachromosomal episomes (and/or, in rare cases, integrates into the host genome on chromosome 19), thereby promoting persistent host cell transgene expression. Among the many available viral gene delivery systems, AAV vectors are attractive candidates because of their ability to mediate efficient transfer and stable expression of therapeutic genes in various tissues, such as the brain, liver, and heart. AAV has several advantages over other viral vectors. First, AAV naturally infects humans, inducing very mild immune responses but not causing disease. Second, recombinant AAV vectors are able to induce long-term transgene expression in non-dividing cells after a single delivery. Third, as a gene therapy vector, AAV is able to target specific tissues or cell types by using specific AAV serotypes. Thirteen AAV serotypes and more than 100 AAV variants have been isolated from human/non-human primate tissues and evaluated in various model systems.