In addition to RPE65, CEP290 (LCA10) is an LCA gene that accounts for ∼20% of LCA patients, making it one of the most frequent causes of LCA.85 The gene encodes a ciliary protein that is required to maintain PR structure and functions.86 Because CEP290 is ∼8 kb in size, the limited packaging capacity of rAVV makes it difficult to develop a gene augmentation approach. The gene discussed is RPE65; the disease is Leber congenital amaurosis.