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DiR-MDS is a preclinical RNA therapeutic being developed by Aptadir Therapeutics for the treatment of Myelodysplastic Syndrome (MDS). It belongs to a novel class of molecules called DNMT1-interacting RNAs (DiRs), which are non-coding RNAs that naturally regulate DNA methylation. DiR-MDS works by binding to and inhibiting DNA methyltransferase 1 (DNMT1), the enzyme responsible for maintaining DNA methylation patterns. By targeting DNMT1 in a locus-specific manner, DiR-MDS aims to prevent the aberrant hypermethylation of tumor suppressor genes, potentially offering a more precise epigenetic therapy compared to global hypomethylating agents.
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