Prime editing for functional repair in patient-derived disease models
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.
- Type: Other
- Archiver: European Genome-Phenome Archive (EGA)
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|Illumina NovaSeq 6000
Prime editing for functional repair in patient-derived disease models.
Nat Commun 11: 2020 5352