A clinical trial is currently underway in which QR-110, an RNA antisense oligonucleotide for intravitreal injection, is being tested in patients with Leber congenital amaurosis carrying the deep-intronic mutation c.2991 + 1655 A > G in the ciliopathy gene centrosomal protein 290 (CEP290) (NCT03140969). Here, CEP290 is linked to Leber congenital amaurosis.