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Updated: Jun 13, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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治疗性基因编辑用于血红蛋白病变.

Ugo Testa1, Giuseppe Leone2, Maria Domenica Cappellini3

  • 1Istituto Superiore Sanità, Roma, Italy.

Mediterranean journal of hematology and infectious diseases
|September 11, 2024
PubMed
概括

基因编辑为治疗状细胞病 (SCD) 和β-血病 (TDT) 开辟了一个新时代. 像CRISPR-Cas9和基编辑这样的先进技术旨在纠正遗传缺陷或提高胎儿血红蛋白以获得有效的治疗方法.

关键词:
基因编辑 基因编辑血球蛋白病变 血球蛋白病变状细胞贫血症是什么血病 (Thalassemia) 是一种疾病.基因治疗的基因疗法

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