Target intelligence / Profile preview

Dystrophia Myotonica Protein Kinase mRNA (DMPK mRNA)

Target
DMPK mRNA
Molecular classification
mRNA, Non-coding RNA
01

Overview

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.

Other names
DM protein kinase mRNADM-PK mRNADM1PK mRNAdystrophia myotonica-protein kinase mRNAmyotonin-protein kinase mRNAMT-PK mRNAMDPK mRNA
02

Mechanism of action

Antisense oligonucleotides to reduce DMPK mRNA levels; small molecules to disrupt RNA foci formation and MBNL interaction; gene silencing strategies

03

Biological functions

Template for protein translationRegulation of alternative splicing (mutant form)Sequestration of RNA-binding proteins (mutant form)
04

Disease associations

Myotonic dystrophy type 1 (DM1)
05

Safety considerations

Off-target effects of antisense oligonucleotidesImmunogenicity of therapeutic agentsDelivery challenges to affected tissues
06

Biomarkers

CTG repeat length in DMPK geneLevels of DMPK mRNAMis-splicing events in target genes

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