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Target intelligence / Profile preview
Dystrophia Myotonica Protein Kinase RNA (DMPK RNA)
- Target
- DMPK RNA
- Molecular classification
- RNA, mRNA, Non-coding RNA (in DM1), Transcript
01
Overview
DMPK RNA is the messenger RNA transcribed from the DMPK gene, which encodes the myotonic dystrophy protein kinase. In myotonic dystrophy type 1 (DM1), expanded CUG repeats within the DMPK mRNA form nuclear foci, sequestering regulatory proteins and disrupting alternative splicing. This toxic gain-of-function mechanism is central to DM1 pathogenesis.
- Other names
- Myotonic Dystrophy Protein Kinase mRNADM Protein Kinase RNAExpanded CUG Repeat RNA
02
Mechanism of action
Expanded CUG repeats in DMPK RNA form nuclear foci, sequestering MBNL proteins and disrupting alternative splicing.
03
Biological functions
mRNA transportRegulation of DMPK protein expressionRegulates muscle contraction
04
Disease associations
Myotonic Dystrophy Type 1 (DM1)
05
Safety considerations
Targeting RNA can have off-target effectsDelivery of therapeutics to specific tissuesPotential for immune response to RNA-based therapies
06
Biomarkers
Expanded CUG repeat length in DMPK mRNAPresence of nuclear RNA fociMBNL protein sequestrationAberrant alternative splicing patterns
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