RPE65 and severe early-childhood-onset retinal dystrophy: Since the development of gene-based therapy for RPE65-associated LCA/EOSRD, there has been considerable interest in novel treatments for other molecular forms of LCA/EOSRD.3, 14 Lentiviral vector gene replacement, antisense oligonucleotide, and CRISPR/Cas9-based techniques are all under active investigation as viable interventions in CEP290 LCA/EOSRD.15, 16, 17, 18, 19