Chiorini, John (Jay) (NIDCR)
Adeno-associated viruses (AAVs) constitute, as a group, the vehicle of choice for gene therapy because of several attractive features. Among others, AAVs are less pathogenic than other viruses, and they can be used for the long-term expression of therapeutic genes.
This invention describes a simple ion-exchange (HPLC) methodology to purify different AAV serotypes. The protocol, which can be readily scaled up, details the efficient concentration of fully infective AAV particles, and is applicable to a number of promising serotypes for which efficient purification methodologies are currently lacking. Significantly, the method consistently produces higher infectivity per particle ratios than standard methods.
This invention, coupled with NIH invention E-325-2001, entitled "Highly Scalable Production of AAV in Insect Cells," would give a licensee a purification system that can be readily scaled-up to efficiently produce recombinant adeno-associated viruses for clinical trial development.