S264: EDIT-301 SHOWS PROMISING PRELIMINARY SAFETY AND EFFICACY RESULTS IN THE PHASE I/II CLINICAL TRIAL (RUBY) OF PATIENTS WITH SEVERE SICKLE CELL DISEASE USING HIGHLY SPECIFIC AND EFFICIENT ASCAS12A ENZYME
S264:EDIT-301 在针对重症镰状细胞病患者的 I/II 期临床试验 (RUBY) 中显示出令人鼓舞的初步安全性和有效性结果,该试验使用了高特异性和高效的 ASCAS12A 酶。
期刊:European Journal of Investigation in Health Psychology and Education
影响因子:2.6
doi:10.3390/ejihpe13080109
Gellisch, Morris; Schäfer, Thorsten; Yahya, Imadeldin; Joswig, Matthias; Cheng, Xin; Morosan-Puopolo, Gabriela; Brand-Saberi, Beate; Hanna, Rabi; Frangoul, Haydar; Mckinney, Christopher; Pineiro, Luis; Mapara, Markus; Chang, Kai-Hsin; Jaskolka, Michael; Kim, Keunpyo; Rizk, Maha; Afonja, Olubunmi; Lawal, Adebayo; Walters, Mark