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

• Published in: Cell stem cell Vol. 31; no. 7; pp. 961 - 973.e8
• Main Authors: Li, Shiqi, Ling, Sikai, Wang, Dawei, Wang, Xiaoyuan, Hao, Fangyuan, Yin, Liufan, Yuan, Zhongtao, Liu, Lin, Zhang, Lin, Li, Yu, Chen, Yingnian, Luo, Le, Dai, Ying, Zhang, Lihua, Chen, Lvzhe, Deng, Dongjie, Tang, Wei, Zhang, Sujiang, Wang, Sanbin, Cai, Yujia
• Format: Journal Article
• Language: English
• Published: Elsevier Inc 05.07.2024
• Subjects: CD34+ cell; gene addition; gene therapy; globin; HSPC; insulator; lentivirus; red blood cell; single-cell DNA sequencing; β0/β0 thalassemia; single-cell DNA sequencing; β0/β0 thalassemia; globin; gene therapy; gene addition; CD34+ cell; insulator; lentivirus; HSPC; red blood cell

β0/β0 thalassemia is the most severe type of transfusion-dependent β-thalassemia (TDT) and is still a challenge facing lentiviral gene therapy. Here, we report the interim analysis of a single-center, single-arm pilot trial (NCT05015920) evaluating the safety and efficacy of a β-globin expression-op...