CEP290 and Leber congenital amaurosis: Currently, one clinical trial is using SaCas9 and delivery by AAV into the retina to treat Type 10 Leber congenital amaurosis (LCA10), a severe retinal dystrophy caused by mutations in the CEP290 gene, by removing the aberrant splice donor created by the IVS26 mutation and thereby restoring normal CEP290 expression 185.