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Target intelligence / Profile preview
Dystrophin pre-mRNA Exon 44 (DMD Exon 44)
- Target
- DMD Exon 44
- Molecular classification
- mRNA, Exon, Pre-mRNA splicing target
01
Overview
Dystrophin pre-mRNA exon 44 is the 44th exon in the pre-mRNA transcript of the DMD gene. It is a therapeutic target for exon skipping strategies in Duchenne muscular dystrophy (DMD). Antisense oligonucleotides (ASOs) are designed to bind to this exon, causing it to be skipped during mRNA splicing, potentially restoring the reading frame and producing a truncated but partially functional dystrophin protein.
- Other names
- DMD exon 44Dystrophin mRNA exon 44Exon 44 of Dystrophin
02
Mechanism of action
Antisense oligonucleotide-mediated exon skipping
03
Biological functions
mRNA splicing regulationDystrophin protein productionMaintenance of muscle fiber integrity (indirect)
04
Disease associations
Duchenne Muscular Dystrophy (DMD)Becker Muscular Dystrophy (BMD)
05
Safety considerations
Off-target effects of ASOsImmune response to ASOsLimited tissue penetration of ASOsLong-term efficacy of exon skipping
06
Interacting drugs
NS-089/NCNP-02
2 more in the full profile.
07
Biomarkers
Dystrophin protein levelsExon 44 skipping efficiencyDMD mRNA levels
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