Improving Site‐Directed RNA Editing In Vitro and in Cell Culture by Chemical Modification of the GuideRNA |
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| Authors: | Paul Vogel Marius F. Schneider Jacqueline Wettengel Dr. Thorsten Stafforst |
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| Affiliation: | Interfaculty Institute of Biochemistry, University of Tübingen, Auf der Morgenstelle 15, 72076 Tübingen (Germany) http://www.ifib.uni‐tuebingen.de/research/stafforst.html |
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| Abstract: | Adenosine‐to‐inosine deamination can be re‐addressed to user‐defined mRNAs by applying phosphothioate/2′‐methoxy‐modified guideRNAs. Dense chemical modification of the guideRNA clearly improves performance of the covalent conjugates inside the living cell. Furthermore, careful positioning of a few modifications controls editing selectivity in vitro and was exploited for the challenging repair of the Factor 5 Leiden missense mutation. |
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