Following the use of AONs to rescue the splice defects in CEP290 associated with the c.2991+1655A>G in humanized transgenic mice, and in cell lines from LCA patients homozygous for this deep-intronic mutation [14,15,16,17], a clinical trial investigating the safety and efficacy of these molecules for the treatment of CEP290-associated LCA has now commenced (NCT03140969 [23]). This evidence concerns the gene CEP290 and Leber congenital amaurosis.