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Target intelligence / Profile preview
Syntaxin-binding protein 1 (Munc18-1)
- Target
- Munc18-1
- Molecular classification
- Vesicle trafficking protein, SM (Sec1/Munc18-like) protein family, Other (does not fall under receptor, enzyme, transporter, ion channel, or transcription factor)
01
Overview
Syntaxin-binding protein 1 (Munc18-1) is a highly conserved peripheral membrane protein essential for synaptic vesicle docking, priming, and fusion at the presynaptic membrane. Encoded by the STXBP1 gene, it regulates the assembly of the SNARE complex by binding syntaxin-1, thereby controlling neurotransmitter release and synaptic function. The protein has a crucial regulatory role in neuronal communication and neurodevelopment, and its dysfunction due to genetic mutation is linked with severe neurodevelopmental and epileptic encephalopathies, as well as several other neuropsychiatric and neurodegenerative conditions. Currently, no therapies directly target this protein; its main clinical relevance lies in genetic disease
- Other names
- Munc18-1STXBP1Sec1 (in yeast)Syntaxin binding protein 1
02
Mechanism of action
Not applicable—no known drugs directly target this protein, though therapies for associated epilepsies are commonly used (generally anti-epileptics not directly targeting Syntaxin-binding protein 1)
03
Biological functions
Synaptic vesicle docking and fusionRegulation of neurotransmitter releaseNeurodevelopment and neurosynaptic plasticityNeuroendocrine secretion (including insulin and thyroxine release)
04
Disease associations
Epileptic encephalopathy (including Ohtahara syndrome, early infantile epileptic encephalopathy)Intellectual disabilityAutism spectrum disorderSchizophreniaParkinson's diseaseAlzheimer's diseaseMultiple sclerosisDuchenne muscular dystrophyAtaxiaNeurodegenerative diseaseMovement disorders
05
Safety considerations
Essential for neuronal viability and neurotransmitter release; loss-of-function states cause severe neurodevelopmental disorders and seizuresPotential on-target toxicity could include impaired synaptic transmission, developmental delay, encephalopathy, and movement disorders if directly inhibited or dysregulated
06
Interacting drugs
None identified as direct, specific interacting drugs currently in clinical use; there is no evidence of FDA-approved drugs directly targeting Syntaxin-binding protein 1 for therapy
07
Biomarkers
STXBP1 mutation (biomarker for diagnosis of STXBP1 encephalopathies and related epileptic syndromes)Likely other genetic biomarkers for patient identification in research or clinical genetics
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