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Article

Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing

5 Dec 2022

Abstract excerpt

Leber congenital amaurosis (LCA), an inherited retinal degeneration, causes severe visual dysfunction in children and adolescents. In patients with LCA, pathogenic variants, such as RPE65 , are evident in specific genes, related to the functions of retinal pigment epithelium and photoreceptors. In contrast to the original Cas9, base editing tools can correct pathogenic substitutions without generation of DNA...

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PMID: 36589710
DOI: 10.1016/j.omtn.2022.11.021

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