RNA binder 2 (Compound 20) serves as a covalent inhibitor of r(CUG)expRNA in myotonic dystrophy type 1 (DM1). It attaches to RNA through the Hoechst scaffold, hindering the binding of MBNL1 and reinstating the normal splicing process. This compound holds potential for research in the context of myotonic dystrophy type 1.
Target-Kategorie:
DNA/RNA Synthesis
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