Chiorini, John (Jay) (NIDCR)
Currently, adeno-associated virus (AAV) represents the gene therapy vehicle of choice because it has many advantages over current strategies for therapeutic gene insertion. AAV is less pathogenic than other virus types; stably integrates into dividing and non-dividing cells; integrates at a consistent site in the host genome; and shows good specificity towards various cell types for targeted gene delivery.
To date, 11 AAV isolates have been isolated and characterized. New serotypes derived from non-human animal species have added to the specificity and repertoire of current AAV gene therapy techniques by avoiding the immunologic complications associated with human isolates.
This invention describes vectors derived from an avian AAV. These vectors have innate properties related to their origin that may confer them with a unique cellular specificity in targeted human gene therapy and a unique immunologic profile that would avoid neutralization by pre-existing antibodies. Therefore, vectors derived from this avian AAV are likely to find novel applications for gene therapy in humans. Furthermore because of their species of origin, this vector would also be useful in the engineering of avian cells.