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Target intelligence / Profile preview
Dystrophin pre-mRNA Exon 50 (DMD Exon 50)
- Target
- DMD Exon 50
- Molecular classification
- RNA, Pre-mRNA, Exon, Splice variant
01
Overview
Dystrophin pre-mRNA exon 50 is a critical exon within the pre-mRNA transcript of the DMD gene. Mutations or deletions affecting this exon often disrupt the reading frame, leading to Duchenne muscular dystrophy. Therapeutic strategies, such as exon skipping using antisense oligonucleotides, aim to restore the reading frame and produce a truncated but partially functional dystrophin protein.
- Other names
- DMD Exon 50Dystrophin exon 50Pre-mRNA exon 50
02
Mechanism of action
Antisense oligonucleotides (AOs) induce exon skipping during pre-mRNA splicing, restoring the reading frame and allowing for the production of a truncated, but partially functional, dystrophin protein.
03
Biological functions
mRNA splicingDystrophin protein synthesisMaintenance of mRNA reading frame
04
Disease associations
Duchenne Muscular Dystrophy (DMD)Becker Muscular Dystrophy (BMD)
05
Safety considerations
Off-target effects of AOsImmune response to AOsVariable efficacy of exon skippingLimited long-term data on efficacy and safety
06
Interacting drugs
Antisense Oligonucleotides (AOs)
1 more in the full profile.
07
Biomarkers
Dystrophin protein expression in muscle biopsiesmRNA levels of skipped exonsSerum creatine kinase levels
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