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Target intelligence / Profile preview
Dystrophin pre-mRNA Exon 45 (DMD Exon 45)
- Target
- DMD Exon 45
- Molecular classification
- RNA, Pre-mRNA, Exon
01
Overview
Dystrophin pre-mRNA exon 45 refers to the 45th exon within the precursor messenger RNA (pre-mRNA) transcribed from the DMD gene, which encodes dystrophin. Dystrophin is a large cytoskeletal protein essential for stabilizing muscle cell membranes, and its absence or dysfunction leads to Duchenne Muscular Dystrophy (DMD). Targeting this exon with antisense oligonucleotides or gene editing tools allows for exon skipping, restoring the reading frame and enabling the production of truncated but partially functional dystrophin in certain DMD patients.
- Other names
- DMD Exon 45 pre-mRNADystrophin mRNA Exon 45Exon 45 of Dystrophin pre-mRNA
02
Mechanism of action
Antisense oligonucleotide-mediated exon skipping
03
Biological functions
mRNA splicingProtein synthesis regulationGene expression
04
Disease associations
Duchenne Muscular Dystrophy (DMD)
05
Safety considerations
Potential for off-target effects of ASOsImmune response to ASOsLimited dystrophin restorationLong-term efficacy and safety of exon skippingDelivery challenges for gene editing approaches
06
Interacting drugs
Casimersen
1 more in the full profile.
07
Biomarkers
Dystrophin protein expression levelsPresence of mutations amenable to exon 45 skipping
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