Recent Phase I clinical trials for RPE65 gene replacement therapy provide hopeful prospects for the treatment of inherited retinal dystrophies (Bainbridge et al., 2008; Hauswirth et al., 2008; Maguire et al., 2008; Cideciyan et al., 2009; Maguire et al., 2009). The gene discussed is RPE65; the disease is inherited retinal dystrophy.