Since completion of the human genome project, RNA has been shown to play crucial roles in nearly all aspects of human biology. Accordingly, aberrant RNA activity has been linked to many human diseases. These connections have made the targeting of RNAs attractive as a novel therapeutic strategy; however, our ability to drug RNA molecules remains challenging. Recent studies have shown that RNAs are invariably bound by RNA-binding proteins (RBPs), which play key roles in regulating many aspects of coding and non-coding RNA biology, including RNA processing and function, nuclear export, cellular localization, and stability. Disruption of this complex network of RNA-protein interactions (RPIs) has been implicated in a number of human diseases, including cancers and neurodegenerative disorders. Thus, the targeting of RBPs and RPIs has arisen as a new frontier in RNA-targeted drug discovery. In this talk, I will discuss our laboratory’s efforts towards the development and application of innovative assay technologies for targeting RPIs to enable the discovery of novel RNA-targeted therapeutics.

Enabling technologies for targeting RNA-protein interactions

ACS Fall 2021

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