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Target intelligence / Profile preview
Dystrophin (DMD)
- Target
- DMD
- Molecular classification
- Cytoskeletal protein, Actin-binding protein, Structural protein
01
Overview
Dystrophin is a large, rod-shaped cytoskeletal protein primarily found in skeletal and cardiac muscle. It plays a critical role in maintaining the structural integrity of muscle fibers by linking the internal cytoskeleton (actin filaments) to the extracellular matrix through the dystrophin-associated protein complex (DAPC). Mutations in the dystrophin gene cause Duchenne and Becker muscular dystrophies, characterized by progressive muscle weakness and degeneration.
- Other names
- DMD protein
02
Biological functions
Sarcolemma stabilizationForce transmission in muscleScaffolding for signaling complexesActin bindingExtracellular matrix linking
03
Disease associations
Duchenne Muscular DystrophyBecker Muscular DystrophyCardiomyopathy
04
Safety considerations
Challenges in gene therapy delivery to muscle tissueImmune response to gene therapy vectorsOff-target effects of exon skipping therapies
05
Biomarkers
Serum creatine kinase (elevated in DMD/BMD)Dystrophin protein levels in muscle biopsy
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