In vivo base editing rescues photoreceptors in a mouse model of retinitis pigmentosa
Jing Su, Kaiqin She, Li Song, Xiu Jin, Ruiting Li, Qinyu Zhao, Jianlu Xiao, Danian Chen, Hui Cheng, Fang Lü, Yuquan Wei, Yang Yang
This study uses base editing to correct a Pde6b mutation in a mouse model of retinitis pigmentosa, restoring photoreceptor function and visual ability.
Molecular Therapy — Nucleic Acids · Feb 14, 2023Peer-reviewedGenetics