Author: Morgan, Brittany S; Forte, Jordan E; Hargrove, Amanda E
Title: Insights into the development of chemical probes for RNA Document date: 2018_9_19
ID: wupre5uj_3
Snippet: While ligands that bind non-ribosomal RNA in vitro have been reported for decades, the development of chemical probes with evidence of specific small molecule:RNA engagement in cell or animal models has dramatically increased in the last four years. Recent studies report several drug-like small molecules that target a range of RNAs in animal models, including riboswitches (15) , miRNAs, (16, 17) splice sites (18) , and mature mRNAs (19) , at leas.....
Document: While ligands that bind non-ribosomal RNA in vitro have been reported for decades, the development of chemical probes with evidence of specific small molecule:RNA engagement in cell or animal models has dramatically increased in the last four years. Recent studies report several drug-like small molecules that target a range of RNAs in animal models, including riboswitches (15) , miRNAs, (16, 17) splice sites (18) , and mature mRNAs (19) , at least one of which is currently in clinical trials (NCT02268552). Multivalent ligands have been reported that target r(CUG) exp repeats of Myotonic Dystrophy Type 1 (DM1) in D. melanogaster (20) and mouse models (21) . These recent successes confirm that selective RNA targeting is achievable in biological systems; however, the limited examples over years of effort highlight the challenges associated with selectively probing RNA.
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