title: Improving site-directed RNA editing in vitro and in cell culture by chemical modification of the guideRNA.
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.
authors.affiliation: Interfaculty Institute of Biochemistry, University of Tübingen, Auf der Morgenstelle 15, 72076 Tübingen (Germany) http://www.ifib.uni-tuebingen.de/research/stafforst.html.
authors.forename: Paul
authors.initials: P
authors.lastname: Vogel
authors.forename: Marius F
authors.initials: MF
authors.lastname: Schneider
authors.forename: Jacqueline
authors.initials: J
authors.lastname: Wettengel
authors.forename: Thorsten
authors.initials: T
authors.lastname: Stafforst
mesh_terms: D018929:Cell Culture Techniques; D003641:Deamination; D012313:RNA / Q000378:metabolism*
chemical_list: D012313:RNA
keywords: RNA editing; antagomirs; genetic diseases; nucleic acid analogues; transcript repair