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Target intelligence / Profile preview
Vacuolar protein sorting 13 homolog C (VPS13C)
- Target
- VPS13C
- Molecular classification
- Lipid transport protein, Membrane contact site protein (organelle tether), Other (large cytoplasmic protein, not classified as receptor or enzyme)
01
Overview
Vacuolar protein sorting 13 homolog C (VPS13C) is a large cytoplasmic lipid transport protein localized at membrane contact sites, particularly between the endoplasmic reticulum (ER) and late endosomes/lysosomes. VPS13C is believed to transfer lipids via a bridge-like mechanism, facilitating bulk lipid delivery between adjacent bilayers, and is required for proper mitochondrial function and lysosomal regulation. Mutations in VPS13C are strongly associated with familial forms of early-onset Parkinson's disease, suggesting a critical role in neurodegenerative disease. The protein interacts with ER and endosomal membranes through specific motifs, mediating organelle tethering and lipid flux. No drugs are currently known to target VPS13C directly, and its main clinical significance lies in genetic diagnostics and research into neurodegenerative pathology.
- Other names
- VPS13CC230055H22RikVacuolar protein sorting 13C
02
Mechanism of action
Not applicable (no approved drugs directly target VPS13C as of current knowledge)
03
Biological functions
Lipid transport between membranesMembrane contact site organizationRegulation of mitophagy (specifically negative regulation of type 2 mitophagy)Maintenance of mitochondrial function and membrane potentialLysosomal function regulation
04
Disease associations
Neurodegenerative disease (notably Parkinson’s disease types 23, autosomal recessive early-onset)Lysosomal dysfunction-related disordersPotential metabolic and mitochondrial diseases (mouse model evidence)
05
Safety considerations
Mutations lead to early-onset Parkinson's disease in humansLysosomal and mitochondrial dysfunction when impaired
06
Biomarkers
Loss-of-function mutations (e.g., for identifying genetic risk in familial Parkinson’s disease)
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