Prime editing for functional repair in patient-derived disease models

Prime editing for functional repair in patient-derived disease models

Prime editing uses Cas9 nickase fused to a reverse transcriptase to edit genetic information. Here, the authors prime edit primary adult stem cells in 3D organoid cultures to show functional correction of pathogenic mutations without genome-wide off-target effects.

Bibliographic Details
Main Authors: Imre F. Schene, Indi P. Joore, Rurika Oka, Michal Mokry, Anke H. M. van Vugt, Ruben van Boxtel, Hubert P. J. van der Doef, Luc J. W. van der Laan, Monique M. A. Verstegen, Peter M. van Hasselt, Edward E. S. Nieuwenhuis, Sabine A. Fuchs
Format: Article
Language:English
Published: Nature Publishing Group 2020-10-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-020-19136-7

Internet

https://doi.org/10.1038/s41467-020-19136-7

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