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Dystrophin pre-mRNA (exon 51)
- Molecular classification
- Precursor mRNA, RNA
01
Overview
Dystrophin pre-mRNA containing exon 51 is a critical molecular target for therapies aiming to treat Duchenne muscular dystrophy caused by out-of-frame mutations involving this region. By inducing targeted skipping of this exon during RNA processing using antisense technologies, it is possible to restore production of a shorter but still functional form of the essential structural protein dystrophin—offering clinical benefit by converting severe disease phenotypes toward milder ones seen in Becker muscular dystrophy patients.
- Other names
- DMD pre-mRNA (exon 51)Dystrophin exon 51 pre-mRNA
02
Mechanism of action
Antisense oligonucleotide-mediated exon skipping to restore dystrophin reading frame.
03
Biological functions
Gene expressionPre-mRNA splicingRegulation of dystrophin production
04
Disease associations
Duchenne Muscular DystrophyBecker Muscular Dystrophy
05
Safety considerations
NephrotoxicityInjection site reactionsLimited long-term efficacy dataImmune response
06
Interacting drugs
Eteplirsen
07
Biomarkers
Dystrophin protein levelsExon skipping efficiencyMuscle function tests
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