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A novel aurone RNA CAG binder inhibits the huntingtin RNA-protein interaction.

Giovanna BallarinMaddalena BiasiottoAnnika ReisbitzerMarlen HegelsMichael BolteSybille KraußDaria V Berdnikova
Published in: RSC medicinal chemistry (2024)
Huntington's disease (HD) is a devastating, incurable condition whose pathophysiological mechanism relies on 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 an aurone scaffold that reduces the level of binding of HTT mRNA to the MID1 protein in vitro . The obtained results introduce aurones as a novel platform for the design of functional ligands for disease-related RNA sequences.
Keyphrases
  • nucleic acid
  • small molecule
  • single cell