Pub. Date : 2020 Jun 5
PMID : 32354745
2 Functional Relationships(s)Download |
Sentence | Compound Name | Protein Name | Organism |
| 1 | Niemann-Pick type C (NPC) disease is primarily caused by mutations in the NPC1 gene and is characterized by the accumulation of unesterified cholesterol and lipids in the late endosomal (LE) and lysosomal (Ly) compartments. | Cholesterol | NPC intracellular cholesterol transporter 1 | Homo sapiens |
| 2 | Given its ability to cross the blood brain barrier, we posit VPA provides a potential mechanism to improve the response to 2-hydroxypropyl-b-cyclodextrin, by restoring a functional NPC1 to the cholesterol managing compartment as an adjunct therapy. | Cholesterol | NPC intracellular cholesterol transporter 1 | Homo sapiens |