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Updated: Feb 4, 2026

Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells
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Published on: March 10, 2023

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功能校正和基因组完整性与双重基编辑的β-thalassemic造血干细胞的功能校正和基因组完整性

Nikoletta Y Papaioannou1, Petros Patsali1, Julia Klermund2,3

  • 1Department of Molecular Genetics Thalassemia, The Cyprus Institute of Neurology & Genetics, Nicosia, Cyprus.

Genome biology
|February 3, 2026
PubMed
概括

双重基编辑器 (BE) 通过增强胎儿血红蛋白,有效地纠正β-血症. 这种基因组编辑方法在最小的基因组改变下显示出显著的治疗潜力,为治疗这种常见的遗传疾病提供了更安全的替代方案.

关键词:
编辑基础编辑这就是CAST-seqq.染色体异常是一种染色体异常.在HSPC中,HSPC可以提供各种服务.在HUDEP-2中使用.多重复合 DNA 编辑泰拉西米亚 泰拉西米亚

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