AAV-based therapies induce an immune response against the viral vector and thus are limited to single dosing. We have initiated a program to study these immune responses and are using both mouse models and human samples from our Muscular Dystrophy clinic. By understanding how the immune system interfaces with AAV, we can develop strategies to overcome immune reactions and possibly re-dose. The immune response is also responsible for unwanted serious adverse events in patients dosed with high levels of AAV, so our laboratory is actively assessing ways to reduce these unwanted side effects.
