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Target intelligence / Profile preview
Dystrophin pre-mRNA (exon 53) (N/A)
- Target
- N/A
- Molecular classification
- RNA, pre-mRNA, Splice target
01
Overview
Dystrophin pre-mRNA containing exon 53 is a crucial molecular target in genetic therapies for Duchenne muscular dystrophy. By inducing skipping of this specific exon during mRNA processing, it becomes possible to restore partial function even when disease-causing mutations disrupt normal translation downstream from this region. This approach underpins several current RNA-based therapeutics aimed at treating subsets of patients with amenable genotypes.
- Other names
- DMD pre-mRNA (exon 53)Dystrophin mRNA exon 53 target
02
Mechanism of action
Exon skipping via steric block of splice sites
03
Biological functions
mRNA processingSplicing regulationGene expression
04
Disease associations
Duchenne Muscular DystrophyGenetic Disorders
05
Safety considerations
Off-target effects of ASOsImmune response to ASOsLimited efficacy due to mutation heterogeneityDelivery challenges to muscle tissue
06
Interacting drugs
Antisense oligonucleotides (ASOs) targeting exon 53
1 more in the full profile.
07
Biomarkers
Dystrophin protein expressionExon skipping efficiencymRNA transcript levelsFor specific ASOs, assessment of target sequence and potential off-target effects
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