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Micro-dystrophin (N/A)
- Target
- N/A
- Molecular classification
- Structural protein, Cytoskeletal protein
01
Overview
Micro-dystrophin is a genetically engineered, truncated version of the full-length dystrophin protein, designed for gene therapy in Duchenne muscular dystrophy (DMD). Its reduced size allows packaging into adeno-associated virus (AAV) vectors for delivery to muscle cells, where it partially restores dystrophin function by stabilizing the sarcolemma and linking the cytoskeleton to the extracellular matrix. While not fully functional like native dystrophin, micro-dystrophin aims to ameliorate muscle disease symptoms and improve muscle force.
- Other names
- Truncated dystrophinMini-dystrophinAAV-delivered dystrophin
02
Mechanism of action
Provides partial dystrophin function by stabilizing the muscle cell membrane and linking the cytoskeleton to the extracellular matrix.
03
Biological functions
Sarcolemma stabilizationActin filament bindingLinkage of cytoskeleton to extracellular matrixPartial restoration of dystrophin function
04
Disease associations
Duchenne muscular dystrophy (DMD)
05
Safety considerations
Immune response to AAV vectorOff-target expressionPotential for cardiac toxicity at high expression levelsLong-term efficacy and safety unknown
06
Biomarkers
Micro-dystrophin expression levels in muscle tissueSerum creatine kinase levelsMuscle function tests (e.g., 6-minute walk test)
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