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Target intelligence / Profile preview
Dystrophin (microdystrophin) (DMD)
- Target
- DMD
- Molecular classification
- Cytoskeletal protein, Structural protein
01
Overview
Dystrophin is a large, rod-shaped cytoplasmic protein essential for muscle function, linking the cytoskeleton to the extracellular matrix. Mutations cause Duchenne and Becker muscular dystrophies. Microdystrophin refers to engineered, shortened versions of dystrophin used in gene therapy, retaining key functional domains while being small enough for AAV delivery. Microdystrophins improve membrane stability and reduce pathology but may not fully prevent disease progression.
- Other names
- DystrophinMicrodystrophin
02
Mechanism of action
Gene therapy to express truncated dystrophin (microdystrophin) to partially restore muscle function.
03
Biological functions
Muscle fiber stabilizationSarcolemma integrityLinkage between cytoskeleton and extracellular matrix
04
Disease associations
Duchenne muscular dystrophy (DMD)Becker muscular dystrophy (BMD)
05
Safety considerations
Immune response to AAV vectorOff-target effects of gene therapyLimited long-term efficacyPotential for incomplete restoration of function
06
Biomarkers
Dystrophin protein levelsMuscle fiber integrityMotor function assessments
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