Recently, Russell et al. [23] conducted a phase 3 randomized controlled trial evaluating the efficacy and safety of gene therapy for patients with RPE65 retinopathy, in which four patients were compound heterozygous for the Arg91Trp mutation (and the mutations IVS7 + 2T > A, Trp402Stop [2 patients] and Arg91Gln, respectively). This evidence concerns the gene RPE65 and retinal disorder.