Intravenous administration of EXG110 resulted in substrate correction in Fabry mice and sustained enzyme activity in non-human primates.
静脉注射 EXG110 可纠正法布里病小鼠的底物异常,并维持非人灵长类动物的酶活性。
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.12.039
Li Shaoyong, Yu Pengfei, Wang Hong, Liu Mingwen, Wang Qingzeng, Li Zhongwan, Chen Shuyuan, He Chentao, Zhan Bryan Yue, Tong Lucas Song, Shou Xinyi, Luo Jingjing, Jia Xiaoyuan, Xin Xin, Hu Wei, Lu Zhihong, Wang Yigang, Ye Guojie, Mao Jianhua, Wu Zhenhua