Prime editing for functional repair in patient-derived disease models
收藏NIAID Data Ecosystem2026-03-13 收录
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资源简介：
Prime editing is a novel genome editing technology using fusion proteins of Cas9-nickase and reverse transcriptase, that holds promise to correct a wide variety of genetic defects. We succeeded in efficient prime editing and functional recovery of disease-causing mutations in patient-derived liver and intestinal stem cell organoids. Whole genome sequencing of did not detect off-target mutations or a mutational signature induced by prime editing.EGA study EGAS00001004611
创建时间：
2021-10-12
