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Dystrophin gene (DMD)
- Target
- DMD
- Molecular classification
- Encodes a protein (dystrophin) that is part of the dystrophin-glycoprotein complex
01
Overview
The DMD gene encodes the dystrophin protein, which is crucial for muscle integrity and function. Mutations in this gene lead to Duchenne and Becker muscular dystrophy by disrupting the dystrophin-glycoprotein complex, which normally connects the cytoskeleton to the extracellular matrix in muscle cells.
- Other names
- BMDDMD_HUMANdystrophin (muscular dystrophy, Duchenne and Becker types)
02
Mechanism of action
Exon skipping, which restores a functional dystrophin protein by skipping over mutated exons, thereby allowing the production of a shorter but still functional dystrophin.
03
Biological functions
Muscle cell structural supportCell signalingAnchoring the extracellular matrix to the cytoskeleton
04
Disease associations
Duchenne muscular dystrophyBecker muscular dystrophyX-linked dilated cardiomyopathy
05
Safety considerations
Ensuring efficacy while minimizing off-target effects in gene therapyManaging potential immune responses in gene therapy
06
Interacting drugs
casimersen (AMONDYS 45)
07
Biomarkers
Levels of dystrophin expressionMutations in the dystrophin gene
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