Recombinant adeno-associated virus vector tropism in human retina

The human retina is an attractive target of AAV-mediated gene therapy, however a major ongoing challenge is that AAV vector tropism within the tissue remains poorly defined. As a result, hit or miss vector selection methods have led to very advanced clinical trials, with resultant data continuing to contradict that obtained from animal models, manifesting most often as lack of therapeutic effect. In this study we hypothesised that human post-mortem retina is a useful model for testing recombinant AAV tropism and can be used to compare AAV vector transduction efficiency.