Exploring How Chemically Diverse Fragments Target a Cryptic RNA Binding Pocket through Base Displacement

  • Riboswitches provide unique opportunities to systematically explore how RNA productively interacts with a broad chemical space
  • Combining in vitro binding, cell-based assays, chemoinformatic modeling and structue-based design, we unmasked a cryptic binding site within the cobalamin riboswitch that was exploited to discover compounds that have affinity exceeding the native ligand
  • Our data demonstrate how a privileged biphenyl-like scaffold effectively targets RNA and how other chemistries can be harnessed to achieve high-affinity interactions