RPE65 and Abnormal retinal morphology: It is characterized by major clinical, genetic, and physiopathological heterogeneity with non syndromic and syndromic forms, variable visual outcomes, and more than 45 disease genes with variable inheritance, pattern of expression, and retinal function.1 The safety and efficacy of AAV-based gene replacement therapy in LCA patients harboring RPE65 mutations have paved the way for treating retinal diseases.2,3,4 However, FDA-approved AAV vector genomes are limited in size.