RPE65 and Leber congenital amaurosis: Ocular gene therapy in animal models and human patients may show functional rescue even though it does not fully stop the degenerative process, as was noted in RPE65 gene therapy clinical trials for Leber congenital amaurosis in which loss of photoreceptors continued despite functional recovery in treated areas.72,73 Hence, suppressing the microglial activation and the associated inflammatory cascade through early therapeutic intervention could yield far-reaching therapeutic benefit on morphological preservation apart from the resolution of structural schisis in the retina.