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Dystrophin Gene Exon 44 pre-mRNA (DMD exon 44 pre-mRNA)
- Target
- DMD exon 44 pre-mRNA
- Molecular classification
- Pre-mRNA, Gene segment
01
Overview
The dystrophin gene exon 44 pre-mRNA refers specifically to the precursor mRNA region encompassing exon 44 within the *DMD* transcript. This region is crucial for therapies aiming at restoring partial function in Duchenne muscular dystrophy by inducing targeted skipping during RNA processing—thereby enabling synthesis of a semi-functional form of dystrophin protein even when certain mutations are present.
- Other names
- DMD Exon 44 pre-mRNADystrophin exon 44 pre-mRNAExon 44 of DMD pre-mRNA
02
Mechanism of action
Antisense oligonucleotides induce exon skipping during pre-mRNA splicing, leading to production of truncated dystrophin protein.
03
Biological functions
mRNA splicing regulationRegulation of dystrophin protein production
04
Disease associations
Duchenne Muscular Dystrophy
05
Safety considerations
Off-target effects of ASOsImmune response to ASOsVariable efficacy depending on mutation and patient characteristics
06
Interacting drugs
PMO44
1 more in the full profile.
07
Biomarkers
Dystrophin protein levelsExon 44 skipping efficiencyMotor function assessments
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