RPE65 and retinitis pigmentosa 1: So far, no treatment possibilities are available for patients with RP or LCA caused by mutations in CRB1. Gene augmentation therapies using adeno-associated viral (AAV) vectors are of emerging interest for retinal dystrophies because of the recent FDA approval of an AAV-mediated gene therapy approach for RP and LCA patients with mutations in the RPE65 gene (Maguire et al., 2019).