文献类型： 外文期刊
第一作者： Cheng, Hongya
作者： Cheng, Hongya;Wang, Xin;Mao, Lin;Ding, Huiqian;Hu, Rongjian;Chen, Dandan;Cheng, Xinyue;Wan, Miaomiao;Ye, Shoudong;Zhang, Baowei;Ban, Qian;Hui, Wenqiao;Kang, Hanyue;Xu, Xiaobin;Liu, Jianlei;Qi, Fei;Begum, Nabila;Lu, Daoqiang;Liu, Dahai;Tseng, Hsian-Rong;Xu, Xiaobin
作者机构：
关键词： Nanosubstrate; Nanoparticle; CBE3; BCL11A; HBG; Gene therapy
期刊名称：NANO TODAY （ 影响因子：10.9； 五年影响因子：13.3 ）
ISSN： 1748-0132
年卷期： 2025 年 61 卷
页码：
收录情况： SCI
摘要： Here, we developed and demonstrated a novel integrative system-Silica Nanorods (SNA) substrate cell capture combined with Supramolecular Nanoparticle (SMNP) delivery mediated CBE base editing (SNA & sdot;SMNP & sdot;CBE)- achieving the synchronization of CD34+HSPCs cell capture and gene editing for beta-hemoglobinopathies. First, in vitro study shows it enables efficient and precise modification of BCL11A promoter in CD34+HSPCs, yielding the highly editing efficiency of 50.4 %, thus making an alternative strategy to conventional immunomagnetic cell separation and electroporation transfection system mediated CBE editing (IMS & sdot;EP & sdot;CBE). Then, we transplanted the edited human CD34+HSPCs into severe combined immunodeficiency (SCID) mice by using intraosseous injection strategy. When compared with conventional IMS & sdot;EP & sdot;CBE methods, our results showed that significantly higher human HBG expression in the bone marrow and peripheral blood of recipient mice, and long-term engraftment, evidenced from similar gene expression profiles to na & iuml;ve CD34+HSPCs at 14 weeks. Conclusively, our integrative system-SNA & sdot;SMNP & sdot;CBE & sdot;intraosseous injection-offers an appealing novel way for the unique potential of gene therapy in the clinic application for beta-hemoglobinopathies patients.
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