Phase 1 pilot study for hemophilia-A: AAV8 vector with prophylactic tacrolimus-glucocorticoid achieves therapeutic FVIII activity
针对A型血友病的I期试点研究:AAV8载体联合预防性他克莫司-糖皮质激素治疗可达到治疗性FVIII活性。
期刊:Signal Transduction and Targeted Therapy
影响因子:52.7
doi:10.1038/s41392-026-02599-3
Liu, Wei; Pei, Xiaolei; Yu, Tenghui; Xu, Bingqi; Dai, Xinyue; Xue, Feng; Chen, Lingling; Wang, Xiaolin; Wang, Yuhua; Chen, Yunfei; Liu, Xiaofan; Fu, Rongfeng; Wang, Wentian; Chi, Ying; Li, Hui Yuan; Yang, Renchi; Zhang, Lei