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Updated: Jan 10, 2026

Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells
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Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells

Published on: September 25, 2019

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通过无模板的CRISPR-Cas9编辑和基因转换对ATAD3A病原体变异的基因特异性校正

Taejeong Bae, Yohan Park, Anel LaGrone

    bioRxiv : the preprint server for biology
    |November 24, 2025
    PubMed
    概括

    通过CRISPR-Cas9基因编辑,可以使用无模板基因转换来纠正人类干细胞中的病原性突变. 这种同源重组途径有效地修复引起疾病的变异,为基因疗法提供了潜在的潜力.

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