Three young adult LCA patients with early onset, severe retinal dystrophy, who had missense mutation in RPE65 gene were administered single subretinal injection of recombinant adeno-associated virus vector that expressed RPE65 complementary DNA (cDNA) in one eye, which had worse visual acuity; the contralateral eye was used as a control. The gene discussed is RPE65; the disease is Retinal dystrophy.