AAV8 VP1

The adeno-associated viruses (AAVs), single-stranded DNA packaging viruses belonging to the Parvoviridae family, are promising vectors for gene delivery. There are over 100 AAV genomic isolates, and 13 human and nonhuman serotypes have been described. To date, no diseases have been associated with AAV infections. Recombinant AAV (rAAV) vectors can package foreign (nonviral) genes, transduce both dividing and nondividing cells, and induce long-term gene expression in nondividing cells. In addition, AAV serotypes have different transduction efficiencies for different tissues dictated by their capsid sequence. These properties make AAVs desirable vectors for therapeutic gene delivery. The AAV gene delivery system has been successfully utilized in several human clinical trials, including the treatment of hemophilia B with rAAV8 vector expressing therapeutic levels of the factor IX protein, due to its advantages in transducing hepatocytes.