Litcius/Paper detail

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

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

2024Cell stem cell77 citationsDOI

Topics & Concepts

BiologyThalassemiaGenetic enhancementSingle CenterGlobinTransfusion therapyGeneVirologyBlood transfusionGeneticsInternal medicineImmunologyMedicineHemoglobinopathies and Related DisordersParvovirus B19 Infection StudiesPrenatal Screening and Diagnostics