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Updated: Jun 5, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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通过在人类造血细胞中进行原始编辑来有效地和在现场纠正血红蛋白恒温弹突变

Congwen Shao1,2, Qing Liu1,2, Jinchao Xu3

  • 1Innovation Center for Diagnostics and Treatment of Thalassemia, Nanfang Hospital, Southern Medical University, Guangzhou, Guangdong 510515, China.

Molecular therapy. Nucleic acids
|December 6, 2024
PubMed
概括

主编辑有效地纠正人体细胞和患者干细胞中的血红蛋白常温泉 (Hb CS) 突变. 这种基因编辑方法显示出治疗Hb CS的治疗潜力,这是一个常见的α-thalassemia突变.

关键词:
不变的春天突变 恒定春天突变在HSPC中,HSPC可以提供各种服务.MT:RNA/DNA编辑 编辑主编辑主要编辑.α - thalassemia 这种疾病的症状是:

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