Prime editing for functional repair in patient-derived disease models

Imre F. Schene, Indi P. Joore, Michal Mokry, Anke H.M. van Vugt, Peter M. van Hasselt, Edward E.S. Nieuwenhuis, Sabine A. Fuchs

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Received Date: 30th May 20

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.

Read in full at bioRxiv.

This is an abstract of a preprint hosted on an independent third party site. It has not been peer reviewed but is currently under consideration at Nature Communications.

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Nature Research, Springer Nature