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Target intelligence / Profile preview
Dystrophin Gene pre-mRNA (DMD pre-mRNA)
- Target
- DMD pre-mRNA
- Molecular classification
- RNA, Pre-mRNA, Gene
01
Overview
The dystrophin gene (DMD) pre-mRNA is the primary transcript of the largest known human gene, located on the X chromosome. It undergoes extensive splicing to generate mature mRNA that encodes the dystrophin protein, essential for muscle cell membrane stabilization. Mutations affecting splicing or sequence integrity of the pre-mRNA cause Duchenne and Becker muscular dystrophies. Therapeutic strategies targeting the pre-mRNA aim to modulate splicing and restore functional dystrophin protein production.
- Other names
- Dystrophin primary transcriptDMD pre-mRNA
02
Mechanism of action
Exon skipping via antisense oligonucleotide binding to splice sites, promoting exclusion of targeted exons during pre-mRNA processing.
03
Biological functions
Precursor for dystrophin mRNA isoformsRegulation of dystrophin protein productionSplicing regulation
04
Disease associations
Duchenne muscular dystrophy (DMD)Becker muscular dystrophy (BMD)
05
Safety considerations
Off-target effects of antisense oligonucleotidesImmune response to therapeutic RNAsLimited efficacy depending on mutation and patient variabilityDelivery challenges to muscle tissue
06
Interacting drugs
Antisense oligonucleotides
1 more in the full profile.
07
Biomarkers
Exon skipping efficiencyDystrophin protein levelsAberrant splicing patternsExon inclusion/exclusion ratios
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