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Updated: Jan 10, 2026

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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通过异质造血干细胞 (HSC) 移植治愈状细胞疾病 向 vivo HSC 基因疗法转移

Rina Kansal1,2

  • 1Molecular Oncology and Genetics, Diagnostic Laboratories, Versiti Blood Center of Wisconsin, Milwaukee, WI 53233, USA.

Genes
|November 27, 2025
PubMed
概括

两种新的基因疗法为状细胞病提供了希望,这是一个日益增长的遗传性血液疾病. 这些治疗方法于2023年底获得批准,涉及血造干细胞 (HSC) 的ex vivo基因编辑,但仍然需要干细胞移植.

关键词:
克里斯普尔是什么意思?克里斯普尔是什么意思?基因疗法的基因治疗.血液学疾病 血液学疾病血液造血干细胞移植的结果是什么?血红蛋白 血红蛋白 是一个骨髓缩性条件的形成儿科 儿科 儿科 儿科二次性瘤的发生.状细胞疾病是一种状细胞疾病.治疗药物 治疗药物

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