Mass spectrometry as a drug discovery platform against RNA targets

Mass spectrometry continues to play an increasingly important role in the drug discovery arena. In many drug discovery and biotechnology environments, mass spectrometry constitutes an integral component of the entire drug discovery and development process, from lead identification to quality control of bulk drug substance. Prominent among emerging drug discovery strategies are those which target cellular RNAs. Antisense drugs elicit a therapeutic response by hybridizing a synthetic oligonucleotide to a single stranded region of a target RNA; alternatively, highly structured regions of RNA represent an interesting class of drug target as the complex structural elements often comprise regulatory or functional domains where small molecules can bind with high affinity and specificity. In this paper, we review work performed within the last few years where mass spectrometry has been used to identify and study molecules that bind to RNA drug targets.