Overcoming immune mediated barriers to successful in vivo gene transfer

Adeno-associated viral (AAV) vector gene therapy has shown promise as a possible curative treatment for a number of inherited diseases. However, immune responses directed against AAV vectors remain a hurdle to the broader use of this gene transfer platform. 
Innate and adaptive immune responses directed against the AAV vector capsid or their components can affect the safety and efficacy of gene transfer in humans, in some cases resulting in significant toxicities.  
This presentation will focus on what is known about pre-existing immunity to AAV in humans, what we learned about vector immunogenicity from preclinical studies and clinical trials and on potential strategies to modulate immune responses in gene transfer.