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

• Main Authors: Dong Hyun Jo (Author), Hyeon-Ki Jang (Author), Chang Sik Cho (Author), Jun Hee Han (Author), Gahee Ryu (Author), Youngri Jung (Author), Sangsu Bae (Author), Jeong Hun Kim (Author)
• Format: Book
• Published: Elsevier, 2023-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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 co...