AAV Discovery

Recombinant adeno-associated viruses are among the most favorable means to deliver therapeutic gene products. Despite their low pathogenicity profiles, vector transgene integration is still a chief concern for rAAV-based gene therapy strategies. We are interested in the molecular mechanisms that underlie “random” low-frequency integration and how specific transgene elements can promote genotoxicity and/or tumorigenesis. Knowledge gained from these studies can be directly applied to alternative vector designs to further reduce or entirely prevent these undesirable outcomes.