| Gene Symbol | CYP46A1 |
| Full Name | Cholesterol 24-Hydroxylase |
| Aliases | CH24H |
| Chromosome | 14 |
| Protein Type | Enzyme |
| Target Class | Enzyme |
| Function | CYP46A1 plays a critical role in cholesterol metabolism, which is intimately connected to Alzheimer's disease pathogenesis through multiple mechanisms: |
| Mechanism of Action | Small molecule activator of cholesterol 24-hydroxylase enhancing brain cholesterol turnover |
| Primary Expression | neurons of the brain that catalyzes the conversion of cholesterol to 24-hydroxycholesterol |
| Druggability | Medium (0.51) |
| Clinical Stage | Approved |
| Pathways | Cholesterol metabolism, Oxysterol synthesis, BBB transport |
| UniProt ID | G3V366 |
| Ensembl ID | ENSG00000146386 |
| GeneCards | CYP46A1 |
| Human Protein Atlas | CYP46A1 |
| Associated Diseases | ALS, Alzheimer, Alzheimer'S Disease, Amyotrophic Lateral Sclerosis |
| Known Drugs/Compounds | EFAVIRENZ, efavirenz, spermidine, efavirenz metabolites, lycibarbarspermidines D, neurotransmitters |
| Interactions | BDNF, APP, ABCA1, APOC1, APOC2, SOAT1 |
| SciDEX Target | View Target Profile (6 clinical trials) |
| SciDEX Hypotheses | CYP46A1 Overexpression Gene Therapy TREM2-Mediated Cholesterol Dysregulation in Microg CYP46A1 Gene Therapy for Age-Related TREM2-Mediate (+1 more) |
| KG Connections | 850 knowledge graph edges |
| Databases | GeneCardsNCBI GeneHPASTRING |