id author title date pages extension mime words sentences flesch summary cache txt cord-285290-l7mnq4yb Warner, Katherine Deigan Principles for targeting RNA with drug-like small molecules 2018-07-06 .txt text/plain 7564 400 49 Here, we discuss principles for discovering small-molecule drugs that target RNA and argue that the overarching challenge is to identify appropriate target structures – namely in disease-causing RNAs that have high information content, and consequently appropriate ligand binding pockets. In our view, successful targeting of RNA in therapeutically useful ways with small molecules requires three distinct components: (1) identification of RNAs and RNA motifs with disease-related function, (2) use of screening approaches and libraries likely to identify drug-like molecules with appropriate pharmacological properties and (3) identification of and focus on RNA motifs with sufficient structural sophistication that make it likely that high affinity and specificity in small-molecule binding can be achieved. (1) a therapeutically compelling RNA target, (2) a screening approach that will identify drug-like lead molecules with appropriate pharmacological properties and (3) the identification of RNA motifs with sufficient information content such that high-specificity and high-potency binding to a high-quality pocket is achieved. ./cache/cord-285290-l7mnq4yb.txt ./txt/cord-285290-l7mnq4yb.txt