Toxicology and dose-ranging studies performed in support of clinical trials for Leber congenital amaurosis resulting from biallelic mutations in GUCY2D (LCA1) included mice with these mutations and wild-type cynomolgus macaques; animals received subretinal injections of AAV5-GRK1-GUCY2D or AAV5-GRK1-GFP, respectively (Table 1A). Here, GRK1 is linked to Leber congenital amaurosis.