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Target intelligence / Profile preview
Hydroxyacid oxidase (glycolate oxidase) 1 (HAO1)
- Target
- HAO1
- Molecular classification
- Enzyme, Oxidoreductase, Peroxisomal protein
01
Overview
Hydroxyacid oxidase (glycolate oxidase) 1 is a peroxisomal enzyme, encoded by the HAO1 gene and primarily expressed in the liver and pancreas, that catalyzes the oxidation of glycolate to glyoxylate as part of glyoxylate metabolism. By facilitating the conversion of glycolate, the enzyme plays a key role in the regulation of oxalate production. Aberrant HAO1 activity can lead to pathological accumulation of oxalate, predisposing to disorders such as primary hyperoxaluria and kidney stones. The enzyme can also oxidize other 2-hydroxy acids with lower catalytic efficiency and is implicated as a potential therapeutic target for diseases related to oxalate metabolism[1][2][6][8].
- Other names
- HAO1Glycolate oxidaseGOXGOX1HAOX1GO
02
Mechanism of action
Enzyme inhibition reduces oxidation of glycolate to glyoxylate, lowering oxalate production and potentially reducing risk of calcium oxalate kidney stones and systemic oxalosis
03
Biological functions
Glyoxylate metabolismGlycine degradationPeroxisomal fatty acid metabolismOxidative stress response
04
Disease associations
Primary hyperoxaluriaCalcium oxalate nephrolithiasis (kidney stones)Potential biomarker or mechanistic contributor in liver cirrhosis and prostatic neoplasms
05
Safety considerations
Off-target inhibition may disrupt peroxisomal metabolism and induce accumulation of glycolate or other 2-hydroxy acidsPotential metabolic disturbances in liver and pancreasRisk of unintended alteration of glycine and oxalate balance
06
Interacting drugs
4-carboxy-5-[(4-chlorophenyl)sulfanyl]-1,2,3-thiadiazole (experimental inhibitor)
1 more in the full profile.
07
Biomarkers
Glycolate (substrate levels used as indirect biomarker)Oxalate (product, used as biomarker of HAO1 activity)HAO1 mRNA/protein (in research settings)
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