CEP290 and Leber congenital amaurosis 10: In 2019, Maeder et al. developed a genome-edited therapy (EDIT-101) to treat Leber congenital amaurosis type 10 (LCA10).306 They used an AAV5 vector loaded with saCas9 and sgRNA targeting the CEP290 mutant intron to deliver this gene-editing system into photoreceptor cells via a subretinal injection to delete or inactivate the mutated intron and restore normal expression of CEP290.