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Pre-mRNA (DMD gene) (DMD pre-mRNA)
- Target
- DMD pre-mRNA
- Molecular classification
- RNA, Precursor mRNA
01
Overview
The pre-mRNA of the DMD gene is the unspliced, primary transcript produced from the DMD (dystrophin) gene. It undergoes extensive and complex splicing to generate mature mRNAs that encode dystrophin protein isoforms. Aberrant splicing can lead to Duchenne or Becker muscular dystrophy. Therapeutic strategies target DMD pre-mRNA to modulate splicing and restore dystrophin production.
- Other names
- DMD primary transcriptDystrophin pre-mRNA
02
Mechanism of action
Modulation of pre-mRNA splicing to restore or modify dystrophin protein production
03
Biological functions
Precursor to dystrophin mRNARegulation of dystrophin isoform expression via alternative splicing
04
Disease associations
Duchenne Muscular Dystrophy (DMD)Becker Muscular Dystrophy (BMD)
05
Safety considerations
Off-target splicing effectsImmune response to oligonucleotidesLimited efficacy of exon-skipping therapies
06
Interacting drugs
Antisense oligonucleotides (exon skipping)
2 more in the full profile.
07
Biomarkers
Exon skipping efficiencyDystrophin protein levelsAberrant splicing patterns
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