A compact base editor rescues AATD-associated liver and lung disease in mouse models
一种紧凑型碱基编辑器可挽救小鼠模型中与 AATD 相关的肝脏和肺部疾病
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.08.040
Jenny Gao; Bamidele, Nathan; Pires-Ferreira, Debora; Zheng, Chunwei; Destefano, April; Cheng, Haoyang; Tang, Qiushi; Cao, Yueying; Xie, Jun; Gao, Guangping; Gruntman, Alisha; Sontheimer, Erik; Flotte, Terence R; Xue, Wen
