Modified lentiviral globin gene therapy for pediatric β0/β0 transfusion-dependent β-thalassemia: A single-center, single-arm pilot trial

作者：Shiqi Li, Sikai Ling, Dawei Wang, Xiaoyuan Wang, Fangyuan Hao, Liufan Yin, Zhongtao Yuan, Lin Liu, Lin Zhang, Yu Li, Yingnian Chen, Le Luo, Ying Dai, Lihua Zhang, Lvzhe Chen, Dongjie Deng, Wei Tang, Sujiang Zhang, Sanbin Wang, Yujia Cai · 发表于：Cell stem cell · 年份：2024 · DOI：10.1016/j.stem.2024.04.021 · 被引用次数：79 · 研究领域：Hemoglobinopathies and Related Disorders、Parvovirus B19 Infection Studies、Prenatal Screening and Diagnostics