Bystander base editing interferes with visual function restoration in Leber congenital amaurosis
Seok‐Hoon Lee, Jun Wu, Dongjoon Im, Gue‐Ho Hwang, You Kyeong Jeong, Hui Jiang, Seok Jae Lee, Dong Hyun Jo, William A. Goddard, Jeong Hun Kim, Sangsu Bae
The paper shows that bystander base editing with ABE8e impairs visual function restoration in LCA model mice by causing structural and functional disruptions in RPE65.
bioRxiv · Oct 23, 2024 · PreprintGenetics