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Updated: May 5, 2026

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通过CRISPR/Cas9介导生成两个同位素CEP290突变的iPSC系

Joana Figueiro-Silva1, Melanie Eschment2, Michelle Mennel3

  • 1Institute of Medical Genetics, University of Zurich, Schlieren, Switzerland.

Stem cell research
|July 23, 2025
PubMed
概括

研究人员创造了新的人类干细胞系,在CEP290基因中发生了突变,这对于理解像Leber先天性黄斑症这样的毛病至关重要. 这些CEP290突变细胞系对于研究乳毛生物学和疾病机制非常有价值.

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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
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