Therapeutic base editing to generate a gain-of-function F9 variant for hemophilia B
利用治疗性碱基编辑技术生成治疗B型血友病的F9功能增强型变体。
期刊:Blood
影响因子:23.1
doi:10.1182/blood.2024027870
Baatartsogt, Nemekhbayar; Kashiwakura, Yuji; Hiramoto, Takafumi; Ito, Rina; Sato, Rikako; Nagao, Yasumitsu; Naruoka, Hina; Takata, Haruka; Hayakawa, Morisada; Batjargal, Khishigjargal; Togashi, Tomoki; Hoshino, Atsushi; Shimizu, Taro; Sato, Yusuke; Ishida, Tatsuhiro; Nureki, Osamu; Ohmori, Tsukasa