Gene augmentation has been used successfully to treat other forms of LCA, like the RPE65-based voretigene neparvovec (Russell et al., 2017), and Kruczek and authors show promising evidence of rescue using gene augmentation to overexpress wild-type CRX in CRX-mutant retinal organoids using adeno-associated viruses (AAVs) (Kruczek et al., 2021). The gene discussed is RPE65; the disease is Leber congenital amaurosis.