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Target intelligence / Profile preview
Muscle skeletal receptor tyrosine-protein kinase MuSK (MUSK)
- Target
- MUSK
- Molecular classification
- Receptor, Receptor tyrosine kinase
01
Overview
Muscle skeletal receptor tyrosine-protein kinase MuSK (MUSK) is a receptor tyrosine kinase that plays an essential role in the formation, maintenance, and function of the neuromuscular junction (NMJ), which is the synapse between motor neurons and skeletal muscle fibers. Activation occurs when neuronal agrin binds to LRP4 on muscle cells; this complex then activates MuSK via phosphorylation. Mutations or autoantibodies against MuSK can cause or contribute to disease, like Congenital myasthenic syndromes and Myasthenia gravis.
- Other names
- Muscle-specific tyrosine-protein kinase receptor
02
Mechanism of action
Not applicable, direct inhibitors/activators not mentioned, but targeting via associated molecules like Agrin or LRP4 is possible.
03
Biological functions
Formation of neuromuscular junctions during developmentMaintenance/stabilization of mature NMJsRegulation of gene expression specific to subsynaptic nucleiClustering/phosphorylation of acetylcholine receptors on muscle fibersRetrograde signaling affecting presynaptic nerve terminal differentiationSignal transduction
04
Disease associations
Congenital myasthenic syndromesMyasthenia gravis
05
Safety considerations
Impaired neuromuscular transmission
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