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Regulation of Hematopoietic Stem Cells01:01

Regulation of Hematopoietic Stem Cells

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All blood and immune cells are produced from the multipotent hematopoietic stem cells (HSCs) by the process of hematopoiesis. However, they all have a limited life span. In addition, many are depleted in immune surveillance or combatting an injury or infection. This makes blood one of the most regenerative tissues. Hematopoiesis helps replenish these blood and immune cells, restoring the body's normal functioning. However, overproduction of blood and immune cells can make them cancerous or...
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CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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在造血干细胞中基因编辑.

Jiaoyang Liao1, Yuxuan Wu2

  • 1Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, China.

Advances in experimental medicine and biology
|January 16, 2024
PubMed
概括

基因编辑工具正在彻底改变为血液疾病治疗的造血干细胞 (HSC) 疗法. 本综述涵盖了HSC中的基因编辑应用,临床进展以及有效治疗的挑战.

科学领域:

  • 血液学 血液学 血液学
  • 分子生物学分子生物学
  • 基因治疗 基因治疗

背景情况:

关键词:
面临的挑战 挑战临床试验中的临床试验.基因编辑 基因编辑在HSC上,HSC是高质量的.展望 展望 展望

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  • 造血干细胞 (HSC) 对于血液系统的功能至关重要,并且可以在活体中进行修改.
  • 基因编辑技术提供精确的细胞遗传修饰,包括HSCs.
  • 结论:

    • HSPC基因编辑对各种遗传疾病具有巨大的治疗潜力.
    • 克服临床翻译方面的挑战是实现这些先进疗法的全部好处的关键.