In vivo adenine base editing corrects newborn murine model of Hurler syndrome
Jing Su, Xiu Jin, Kaiqin She, Yi Liu, Li Song, Qinyu Zhao, Jianlu Xiao, Ruiting Li, Hongxin Deng, Fang Lü, Yang Yang
This study corrects a mutation in a mouse model of Hurler syndrome using in vivo adenine base editing, showing reduced GAG accumulation and neurobehavioral deficits.
Molecular Biomedicine · Feb 22, 2023Peer-reviewedGenetics