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Updated: Jul 27, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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精密编辑作为治疗方法用于β-血红蛋白病变

Kiriaki Paschoudi1,2, Evangelia Yannaki2,3, Nikoletta Psatha1

  • 1Department of Genetics, Development and Molecular Biology, School of Biology, Aristotle University of Thessaloniki, 54124 Thessaloniki, Greece.

International journal of molecular sciences
|June 10, 2023
PubMed
概括

基因编辑通过重新激活胎儿血红蛋白 (HbF) 来为状细胞疾病和血病等β-血红蛋白病提供了有希望的治疗方法. 这种先进的治疗方法现在正在临床试验中,对患者显示出积极的初始结果.

关键词:
这就是CRISPR/Cas9的作用.基础编辑 基础编辑基因组编辑 基因组编辑血红蛋白病变 血红蛋白病变 血红蛋白病变胎儿血红蛋白的遗传性持续性主编辑主要编辑.状细胞疾病是一种状细胞疾病.泰拉塞米亚症是一种疾病.

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