RPE65 and Leber congenital amaurosis: With the same idea to validate the safety of LV use for gene therapy clinical trials, but this time to treat RPE65-related Leber congenital amaurosis, we subretinally injected LV-RPE65 (2 doses tested: 2.8 × 105 IU and 2.8 × 106 IU in 100 μL) in healthy NHPs [74].