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Dystrophia myotonica protein kinase (DMPK) mRNA is the messenger RNA transcribed from the DMPK gene, which encodes a serine/threonine protein kinase. In Myotonic Dystrophy Type 1 (DM1), expanded CTG repeats in the 3'-UTR of DMPK mRNA cause a toxic gain-of-function, leading to sequestration of RNA-binding proteins like MBNL and widespread mis-splicing events. Therapeutic strategies aim to reduce toxic DMPK mRNA levels or disrupt its interaction with MBNL.
Antisense oligonucleotides to reduce DMPK mRNA levels; small molecules to disrupt RNA foci formation and MBNL interaction; gene silencing strategies
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