IPRD funded research by Jarrod Mousa, Ph.D.

Gene therapies for rare childhood diseases often use AAV vectors, which act like delivery vehicles carrying healthy genes into cells. A major challenge is that many people already have antibodies that block AAV, and treatment itself can trigger new antibodies that prevent future doses. This project studies how human antibodies attach to AAV particles at the molecular level. Using advanced electron microscopy, the team will map exactly where antibodies bind on the virus surface. These maps will help scientists redesign AAV vectors so they can avoid the immune system, making gene therapy safer and available to more children who need it.