Enabling Technologies for Revealing Druggable Paths in RNA Biology

Following completion of the human genome project, it was revealed that only ~2% of our genome encodes for proteins, and the overwhelming majority is comprised of often highly conserved non-coding RNAs. Since that time, RNA has been shown to significantly impact nearly all of human biology from transcriptional regulation to splicing, translation, RNA function, and catalysis. Accordingly, we have witnessed an explosion in discoveries connecting these RNAs with human diseases, making the search for RNA biology-targeted therapeutics ever more pressing. This presentation will describe some of the new technologies that my group has developed for identifying small molecule inhibitors affecting non-coding RNA biology, as well as new biological discovery platforms for revealing novel druggable pathways in RNA biology.