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Target intelligence / Profile preview
Dystrophin pre-mRNA exon 53 (DMD exon 53)
- Target
- DMD exon 53
- Molecular classification
- RNA, Pre-mRNA, Exon
01
Overview
Dystrophin pre-mRNA exon 53 is a region within the precursor messenger RNA (pre-mRNA) transcribed from the DMD gene. It is a target for exon skipping therapies aimed at restoring the reading frame in patients with Duchenne muscular dystrophy (DMD) who have mutations amenable to skipping this exon, allowing the production of a truncated but partially functional dystrophin protein.
- Other names
- DMD exon 53Duchenne muscular dystrophy exon 53Dystrophin mRNA exon 53
02
Mechanism of action
Antisense oligonucleotides bind to pre-mRNA, masking exon 53 from the splicing machinery, resulting in exon skipping and a shorter, partially functional dystrophin protein.
03
Biological functions
mRNA splicingProtein synthesisMaintenance of muscle fiber integrity (indirectly)
04
Disease associations
Duchenne muscular dystrophy (DMD)Becker muscular dystrophy (BMD)
05
Safety considerations
Renal toxicity (observed with some AONs)Injection site reactionsLimited long-term efficacy dataImmunogenicity
06
Interacting drugs
Golodirsen (Vyondys 53)
1 more in the full profile.
07
Biomarkers
Dystrophin protein levelsExon skipping efficiency in muscle tissueFor patient selection: specific DMD mutations amenable to exon 53 skipping
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