Improving Site‐Directed RNA Editing In Vitro and in Cell Culture by Chemical Modification of the GuideRNA
- 28 May 2014
- journal article
- research article
- Published by Wiley in Angewandte Chemie International Edition
- Vol. 53 (24) , 6267-6271
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.Keywords
This publication has 41 references indexed in Scilit:
- RNase H-mediated degradation of toxic RNA in myotonic dystrophy type 1Proceedings of the National Academy of Sciences, 2012
- Cellular Uptake and Intracellular Trafficking of Antisense and siRNA OligonucleotidesBioconjugate Chemistry, 2011
- Predicting sites of ADAR editing in double-stranded RNANature Communications, 2011
- Functions and Regulation of RNA Editing by ADAR DeaminasesAnnual Review of Biochemistry, 2010
- A large-scale chemical modification screen identifies design rules to generate siRNAs with high activity, high stability and low toxicityNucleic Acids Research, 2009
- Design and delivery of antisense oligonucleotides to block microRNA function in cultured Drosophila and human cellsNature Protocols, 2008
- miR-122 targeting with LNA/2′-O-methyl oligonucleotide mixmers, peptide nucleic acids (PNA), and PNA–peptide conjugatesRNA, 2007
- Position-specific chemical modification of siRNAs reduces “off-target” transcript silencingRNA, 2006
- Silencing of microRNAs in vivo with ‘antagomirs’Nature, 2005
- A general method for the covalent labeling of fusion proteins with small molecules in vivoNature Biotechnology, 2002