| Literature DB >> 24890431 |
Paul Vogel1, Marius F Schneider, Jacqueline Wettengel, Thorsten Stafforst.
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.Entities:
Keywords: RNA editing; antagomirs; genetic diseases; nucleic acid analogues; transcript repair
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Year: 2014 PMID: 24890431 DOI: 10.1002/anie.201402634
Source DB: PubMed Journal: Angew Chem Int Ed Engl ISSN: 1433-7851 Impact factor: 15.336