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Updated: Sep 12, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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针对高效的CRISPR/AAV中介基因组编辑和人类造血干细胞和原始细胞的红细胞分化方案

Devesh Sharma1, Roshani Sinha1, Benjamin J Lesch1

  • 1Department of Surgery, University of California, San Francisco, San Francisco, CA 94143, USA; Eli & Edythe Broad Center for Regeneration Medicine, University of California, San Francisco, San Francisco, CA 94143, USA.

STAR protocols
|August 7, 2025
PubMed
概括

这项研究详细介绍了一种CRISPR-Cas9基因组编辑协议,用于人类造血干细胞和原生细胞 (HSPC). 该方法使用腺相关病毒 (AAV) 以获得高编辑效率和细胞活力,这对于干细胞疗法至关重要.

关键词:
生物技术和生物工程克里斯普尔是什么意思?克里斯普尔是什么意思?细胞生物学 细胞生物学细胞分化 细胞分化细胞培养培养的细胞培养.流动细胞计量 (Flow Cytometry) 是一种流动细胞计.分子生物学分子生物学干细胞是干细胞.

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CRISPR01:59

CRISPR

Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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