Gene therapy is beginning to show promise for the treatment of a variety of inherited ocular diseases, including the first U.S. Food and Drug Administration-approved retinal gene therapy, Luxturna® (voretigene neparvovec), for patients with IRD caused by pathological biallelic RPE65 mutations.32 BCD is an ideal target for retinal gene therapy, but few studies have presented preclinical data to justify its prime candidacy for a gene augmentation strategy, partly due to the lack of well validated in vitro and in vivo models. Here, RPE65 is linked to Bietti crystalline dystrophy.