A novel aurone RNA CAG binder inhibits the huntingtin RNA–protein interactionElectronic supplementary information (ESI) available: Synthetic procedures and characterization of novel compounds, 1H and 13C NMR spectra, single-crystal X-ray analysis data, and description of biological experiments. CCDC 2335536and 2335537. For ESI and crystallographic data in CIF or other electronic format see DOI: https://doi.org/10.1039/d4md00403e

Autor: Ballarin, Giovanna, Biasiotto, Maddalena, Reisbitzer, Annika, Hegels, Marlen, Bolte, Michael, Krauß, Sybille, Berdnikova, Daria V.
Zdroj: MedChemComm; 2024, Vol. 15 Issue: 9 p3092-3096, 5p
Abstrakt: 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.
Databáze: Supplemental Index