RPE65 and Leber congenital amaurosis 2: While an important medical advancement, rAAV vectors currently used in the clinic use capsids of natural serotypes which pose several limitations on efficiency, as seen in clinical trials for Leber’s congenital amaurosis (LCA2) patients (Bainbridge et al, 2008; Hauswirth et al, 2008; Maguire et al, 2008) where photoreceptor degeneration continued even after subretinal gene therapy with an rAAV2‐RPE65 vector (Cideciyan et al, 2013; Bainbridge et al, 2015; Jacobson et al, 2015).