Current ongoing trials in patients affected with Duchenne muscular dystrophy have shown that systemic delivery of 2′-OMePS SSOs on a short-term basis is well tolerated.30,31,32 Systemic administration of 2′-OMePS SSOs to correct the abnormal splicing resulting from the CEP290 c.2991+1655A>G is hampered by the very efficient blood–retina barrier. Here, CEP290 is linked to Duchenne muscular dystrophy.