Despite extraordinary progress achieved in the development of therapies for these diseases over the last decade, including approved gene therapy for RPE65-related Leber congenital amaurosis (LCA) and current clinical trials for certain recessive IRDs, the majority of IRDs remain incurable.2 The development of gene editing techniques based on clustered regularly interspaced short palindromic repeats (CRISPR) has made it possible to correct a wide range of genetic mutations. This evidence concerns the gene RPE65 and Leber congenital amaurosis.