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Target intelligence / Profile preview
Niemann-Pick C1 protein binding inhibition
- Molecular classification
- Membrane protein, Lipid transporter
01
Overview
Inhibition of the binding function of Niemann-Pick C1 (NPC1) protein, a transmembrane protein located in late endosomes and lysosomes that mediates the export of LDL-derived cholesterol. Inhibition leads to intracellular cholesterol accumulation and is implicated in Niemann-Pick disease type C and viral infections. Targeting binding at key domains can modulate cellular lipid trafficking pathways.
02
Mechanism of action
Inhibition of cholesterol binding and transport by Niemann-Pick C1 protein.
03
Biological functions
Cholesterol transportLipid traffickingEndosomal/lysosomal function
04
Disease associations
Niemann-Pick disease type CNeurodegenerative diseaseLysosomal storage disorder
05
Safety considerations
Potential disruption of normal lipid metabolismNeurotoxicity
06
Interacting drugs
U18666A
07
Biomarkers
Cholesterol accumulation in cellsNPC1 mutations
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