RNA as a target for small-molecule therapeutics

Natural antibiotics that specifically target RNA components of the bacterial ribosome set precedence for RNA-directed small-molecule drugs. Structured domains of functional RNAs in bacteria and viruses have thus attracted attention as targets for the discovery of novel anti-infectives. Whereas a growing number of RNA-directed ligands have been reported in the literature, creating synthetic molecules that combine high affinity with selectivity for a specific RNA target remains a key challenge. Ongoing discovery efforts towards this goal, combined with the increasing knowledge of the factors that govern small-molecule recognition of RNA folds, will prepare the ground for the development of novel RNA-targeted therapeutics.