A novel aurone RNA CAG binder inhibits the Huntingtin RNA–protein interaction

Abstract

The Huntington's disease (HD) is a devastating, incurable condition whose pathophysiological mechanism relies on the mutant RNA CAG repeat expansions. Aberrant recruitment of RNA-binding proteins by mutant CAG hairpins contributes to the progress of neurodegeneration. In this work, we identified a novel binder based on the aurone scaffold that reduces the level of binding of HTT mRNA to MID1 protein in vitro. The obtained results introduce aurones as a novel platform for the design of functional ligands for disease-related RNA sequences.