The selective targeting of RNA with small molecules have been the focus of research in the cellular roles of macromolecules. Screening followed by synthetic optimization is likely to provide small molecules that bind in vitro to the intended RNA target with low- to submicromolar binding affinities. There are a variety of RNA-binding assays that are available to determine the strength of a RNA-ligand interaction in addition to biophysical experiments that provide additional information about the binding site and the kinetics/thermodynamics of binding. However, there are still challenges to overcome in order to turn the therapeutic potential of RNA ligands into reality. There will be a requirement for high-throughput screens, the detailed characterization of small molecule-RNA interactions, exploitation of additional RNA targets, RNA ligands with activity in cell culture and in vivo.
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