A precision gene-engineered B cell medicine producing sustained levels of active factor IX for hemophilia B therapy
一种精准基因工程改造的B细胞药物，可产生持续水平的活性凝血因子IX，用于治疗B型血友病。
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.09.001
Liu, Hanlan; Singh, Swati; Mullen, Timothy J; Bullock, Caroline; Keegan, Sean; Patterson, Troy; Thakur, Sakshisingh; Lundberg, Amy; Shenker, Sol; Couto, Ryan; Yadav, Charuta; Dastagir, Shamael; Li, Lily; Bainter, Wayne; Liberzon, Ella; Malloy, Connor R; Lazzarotto, Cicera R; Ohsumi, Toshiro K; Chilakala, Shalini; Chen, Huei-Mei; Kshirsagar, Rashmi; Hohmann, Anja F; Arlauckas, Sean P; Lazorchak, Adam; Scull, Chris; Morgan, Richard A
