RPE65 and Leber congenital amaurosis: AAV-mediated gene therapies have been successfully applied to a number of monogenic retinal dystrophies, including RPE65-associated Leber congenital amaurosis which has received Food and Drug Administration (FDA) approval, and choroideremia and RPGR-associated X-linked retinitis pigmentosa which are in advanced clinical trials [9,10,11,12,13,14].