Target intelligence / Profile preview

Carnitine palmitoyltransferase (CPT)

Target: CPT
Molecular classification: Enzyme, Transferase

Overview

Carnitine palmitoyltransferases are mitochondrial enzymes essential for the transport and metabolism of long-chain fatty acids, playing a critical role in cellular energy production through β-oxidation. Deficiencies in these enzymes lead to severe metabolic diseases primarily affecting liver function during periods requiring increased fat utilization.

Other names: CPT1ACPT1BCPT1CCPT2

Mechanism of action

Catalyzes the reversible transfer of long-chain fatty acyl groups between coenzyme A (CoA) and carnitine, facilitating the transport of activated long-chain fatty acids from the cytosol into the mitochondrial matrix for β-oxidation.

Biological functions

β-oxidation of long-chain fatty acidsRegulation of intracellular CoA/acyl-CoA ratiosFatty acid transport

Disease associations

CPT I DeficiencyCPT II DeficiencyMetabolic disordersHypoketotic hypoglycemiaHepatic encephalopathy

Safety considerations

Hypoketotic hypoglycemiaHepatomegalyHepatic encephalopathyLethargySeizuresComaSudden death (if untreated)

Interacting drugs

Malonyl-CoA

Biomarkers

Elevated carnitine (in newborn screening)

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