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Target intelligence / Profile preview
Hydroxyacid oxidase 1 (glycolate oxidase) (HAO1)
- Target
- HAO1
- Molecular classification
- Enzyme, Oxidoreductase, Peroxisomal protein
01
Overview
Hydroxyacid oxidase 1 (glycolate oxidase, HAO1) is a peroxisomal enzyme predominantly expressed in the liver, where it catalyzes the FMN-dependent oxidation of glycolate to glyoxylate and glyoxylate to oxalate, generating hydrogen peroxide in the process. Mutations or dysregulation in glyoxylate pathway enzymes lead to pathological oxalate accumulation, contributing to conditions like primary hyperoxaluria type 1, characterized by kidney stones and renal injury. The HAO1 mRNA can be selectively targeted by siRNAs or antisense oligonucleotides in therapies designed to reduce harmful oxalate production.
- Other names
- Glycolate oxidaseGOGOXGOX1HAOX1(S)-2-hydroxy-acid oxidase
02
Mechanism of action
Drugs targeting HAO1 primarily act through RNA interference (siRNA-mediated knockdown of HAO1 mRNA) or antisense-mediated degradation of HAO1 mRNA. These mechanisms lead to a reduction in the HAO1 protein levels, thereby inhibiting glycolate oxidase activity.
03
Biological functions
Metabolism of glycolate to glyoxylate and oxalateHydrogen peroxide production via peroxisomal metabolismGlyoxylate detoxification
04
Disease associations
Primary hyperoxaluria type 1 (PH1)Kidney stone formation (hyperoxaluria, nephrolithiasis)
05
Safety considerations
Potential metabolic disturbances if related isoenzymes are affectedHepatic side effects possible due to peroxisomal enzyme inhibitionLong-term enzyme inhibition may alter glyoxylate and glycolate homeostasis
06
Interacting drugs
Lumasiran (an siRNA drug targeting HAO1 mRNA)
1 more in the full profile.
07
Biomarkers
Glycolate levels in blood/urine (pharmacodynamic marker for HAO1 inhibition)Oxalate levels in urine (efficacy monitoring in hyperoxaluria therapy)
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