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paper

Prime editing for functional repair in patient-derived disease models.

📄 Paper Details
Prime editing for functional repair in patient-derived disease models.
["Schene I", "Joore I", "Oka R", "Mokry M", "van Vugt A", "van Boxtel R", "van der Doef H", "van der Laan L", "Verstegen M", "van Hasselt P"]
Nature communications PubMed DOI
Abstract

Prime editing is a recent genome editing technology using fusion proteins of Cas9-nickase and reverse transcriptase, that holds promise to correct the vast majority of genetic defects. Here, we develop prime editing for primary adult stem cells grown in organoid culture models. First, we generate precise in-frame deletions in the gene encoding β-catenin (CTNNB1) that result in proliferation independent of Wnt-stimuli, mimicking a mechanism of the development of liver cancer. Moreover, prime edit...

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