生物
地中海贫血
遗传增强
单中心
珠蛋白
输血疗法
基因
病毒学
输血
遗传学
内科学
免疫学
医学
作者
S. Li,Sikai Ling,Dawei Wang,Xiao-Yuan Wang,Hao Fu,Liufan Yin,Zhongtao Yuan,Lin Liu,Lin Zhang,Yu Li,Yingnian Chen,Le Luo,Ying Dai,Lihua Zhang,Lvzhe Chen,Dan Deng,Wei Tang,Sujiang Zhang,Sanbin Wang,Yujia Cai
标识
DOI:10.1016/j.stem.2024.04.021
摘要
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-optimized and insulator-engineered lentivirus-modified cell product (BD211) in β0/β0 TDT. Two female children were enrolled, infused with BD211, and followed up for an average of 25.5 months. Engraftment of genetically modified hematopoietic stem and progenitor cells was successful and sustained in both patients. No unexpected safety issues occurred during conditioning or after infusion. Both patients achieved transfusion independence for over 22 months. The treatment extended the lifespan of red blood cells by over 42 days. Single-cell DNA/RNA-sequencing analysis of the dynamic changes of gene-modified cells, transgene expression, and oncogene activation showed no notable adverse effects. Optimized lentiviral gene therapy may safely and effectively treat all β-thalassemia.
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