Recent phase I/II clinical trials have demonstrated the safety and efficacy of gene augmentation using adeno-associated viral vectors in patients with Leber Congenital Amaurosis due to RPE65 gene mutations (Bainbridge et al., 2008; Cideciyan et al., 2008, 2009a,b; Hauswirth et al., 2008; Maguire et al., 2008, 2009; Simonelli et al., 2010). The gene discussed is RPE65; the disease is Leber congenital amaurosis.