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Updated: Jun 21, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

Published on: August 9, 2022

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基因治疗和基因编辑策略在遗传性血液疾病中

Xuemei Song1, JinLei Liu1, Tangcong Chen1

  • 1Institute of Blood Diseases, Department of Hematology, Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital, School of Medicine of University of Electronic Science and Technology of China, Chengdu, Sichuan 610000, China.

Journal of genetics and genomics = Yi chuan xue bao
|July 10, 2024
PubMed
概括

基因疗法,包括基于CRISPR的工具,如基因和原始编辑,对遗传性血液疾病有很大的希望. 已批准的疗法针对状细胞病和血病等疾病的BCL11A.

关键词:
基因编辑 基因编辑基因治疗是一种基因疗法.遗传性血液疾病 遗传性血液疾病治疗目标 治疗目标在β-血球蛋白病变中.

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