In vivo genome editing using 244-cis LNPs and low-dose AAV achieves therapeutic threshold in hemophilia A mice
利用244-cis LNP和低剂量AAV进行体内基因组编辑,在A型血友病小鼠中达到了治疗阈值。
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2023.102050
Han, Jeong Pil; Lee, Yeji; Lee, Jeong Hyeon; Chung, Hye Yoon; Lee, Geon Seong; Nam, Yu Ri; Choi, Myeongjin; Moon, Kyoung-Sik; Lee, Haeshin; Lee, Hyukjin; Yeom, Su Cheong