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Updated: Jul 3, 2025

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针对肌纤维细胞肌病的个性化基因特异性CRISPR-Cas9策略 6

Jun Wan Shin1,2, Kyung-Hee Kim1,2, Yukyeong Lee1,2

  • 1Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.

medRxiv : the preprint server for health sciences
|February 14, 2024
PubMed
概括

这项研究开发了个性化的CRISPR-Cas9基因编辑,以非激活肌纤维肌病6 (MFM6) 中突变的BAG3基因. 这种方法显示出创造患者特异性干细胞用于再生医学疗法的前景.

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相关概念视频

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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