Molecular Classification
Enzyme, Acyltransferases
Other Names
Carnitine acyltransferase I, CPTI, CAT1, CoA:carnitine acyl transferase (CCAT), palmitoylCoA transferase I
Disease Roles
Deficiency disordersHypoketotic hypoglycemiaCancer

Carnitine palmitoyl transferase 1 Overview

Carnitine palmitoyl transferase 1 (CPT1) is a mitochondrial enzyme critical for fatty acid metabolism, particularly the transport and oxidation of long-chain fatty acids. It catalyzes the rate-limiting step in the transport of long-chain fatty acids into mitochondria for β-oxidation by transferring the acyl group from long-chain fatty acyl-CoA to L-carnitine to form acylcarnitines. There are three known isoforms: CPT1A (liver), CPT1B (muscle and heart), and CPT1C (neurons). CPT1 is regulated by malonyl-CoA. Deficiency of CPT1 impairs the body’s ability to use certain fats for energy, leading to hypoketotic hypoglycemia and other severe symptoms. Overexpression of CPT1A has been observed in several cancers, supporting tumor cell survival through enhanced fatty acid oxidation.

Mechanism of Action

Inhibition of CPT1 leads to reduced ATP production and cell cycle arrest in cancer cells.

Biological Functions

Fatty acid metabolism
Transport of long-chain fatty acids
β-oxidation
Energy production

Disease Associations

Deficiency disorders
Hypoketotic hypoglycemia
Cancer
Metabolic disorders
Hepatic encephalopathy

Safety Considerations

  • Hypoketotic hypoglycemia
  • Hepatomegaly
  • Hepatic encephalopathy
  • Nervous system damage
  • Coma
  • Sudden death

Associated Biomarkers

Biomarker
Carnitine levels
CPT1A expression