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Annu Rev Pharmacol Toxicol. 2016;56:123-40. doi: 10.1146/annurev-pharmtox-010715-103910. Epub 2015 Oct 22.

Approaches to Validate and Manipulate RNA Targets with Small Molecules in Cells.

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Department of Chemistry, The Scripps Research Institute, Jupiter, Florida 33458; email: ,


RNA has become an increasingly important target for therapeutic interventions and for chemical probes that dissect and manipulate its cellular function. Emerging targets include human RNAs that have been shown to directly cause cancer, metabolic disorders, and genetic disease. In this review, we describe various routes to obtain bioactive compounds that target RNA, with a particular emphasis on the development of small molecules. We use these cases to describe approaches that are being developed for target validation, which include target-directed cleavage, classic pull-down experiments, and covalent cross-linking. Thus, tools are available to design small molecules to target RNA and to identify the cellular RNAs that are their targets.


Inforna; RNA; antibacterials; antisense oligonucleotides; genetic disease; nucleic acids; orphan disease; rational design; target identification

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