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Glyceronephosphate O-acyltransferase (GNPAT)
- Target
- GNPAT
- Molecular classification
- Enzyme, Peroxisomal membrane protein
01
Overview
Glyceronephosphate O-acyltransferase (GNPAT) is a peroxisomal membrane enzyme encoded by the GNPAT gene and is crucial for the first step of ether phospholipid (plasmalogen) biosynthesis. Plasmalogens are specialized lipids found throughout cell membranes, especially in the nervous system (myelin) and other tissues. The enzyme catalyzes the acylation of dihydroxyacetone phosphate, enabling subsequent biosynthetic steps leading to plasmalogen formation[2][4][5][7]. Clinically, GNPAT deficiency (due to genetic mutation) causes rhizomelic chondrodysplasia punctata type 2, a severe developmental disorder, while increased expression or amplification of GNPAT has been correlated with progression and poor prognosis in hepatocellular carcinoma, highlighting its emerging potential as a therapeutic target in cancer biology[3][6].
- Other names
- Dihydroxyacetone phosphate acyltransferaseDHAPATDAPATDAP-ATDHAP-ATAcyl-CoA:dihydroxyacetonephosphate acyltransferaseRCDP2
02
Mechanism of action
no clinically established drugs; experimental inhibition of GNPAT/DRP1 attenuates lipid metabolism and hepatocarcinogenesis
03
Biological functions
Ether phospholipid biosynthesis (plasmalogen synthesis)Lipid metabolismCell membrane formation and maintenanceNervous system maintenance (myelin component)
04
Disease associations
Rhizomelic chondrodysplasia punctata type 2 (RCDP2)Cancer (hepatocellular carcinoma/liver cancer progression)Possibly neurodegenerative conditions (due to roles in myelin and neural lipids)
05
Safety considerations
Deficiency or loss-of-function mutation results in severe reduction of plasmalogens and dysfunctional peroxisomal metabolism, leading to profound developmental defectsDisruption may result in impaired neural and skeletal development
06
Biomarkers
GNPAT gene mutation for rhizomelic chondrodysplasia punctata type 2 (genetic diagnosis)GNPAT amplification/upregulation as a biomarker for poor prognosis in liver cancer
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