LCA patients with CEP290 mutations have a neonatal-onset severe panretinal loss of photoreceptors.10,36 While patients retain a central retinal zone of photoreceptors and an intact visual cortex for up to three decades, it has been suggested from gene augmentation therapeutic trials that the earliest the treatment, the better the effect.36,37,38 Conversely, to subretinal administration which is limited central retina to avoid severe retinal detachment, intravitreal injections of 2'-OMePS seems suited for panretinal treatment. Here, CEP290 is linked to Leber congenital amaurosis.