Prime 编辑在功能上纠正人类类器官和气道上皮细胞中引起囊性纤维化的 CFTR 突变。
关键词: CRISPR DETECTOR cystic fibrosis gene editing human nasal epithelial cells machine learning patient-derived organoids prime editing
Mesh : Humans Cystic Fibrosis Transmembrane Conductance Regulator / genetics metabolism Cystic Fibrosis / genetics pathology metabolism Organoids / metabolism Gene Editing / methods Epithelial Cells / metabolism Mutation / genetics HEK293 Cells CRISPR-Cas Systems / genetics
来 源:
DOI:10.1016/j.xcrm.2024.101544
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