Target intelligence / Profile preview

Carnitine palmitoyl transferase 1 (CPT1)

Target
CPT1
Molecular classification
Enzyme, Acyltransferases
01

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.

Other names
Carnitine acyltransferase ICPTICAT1CoA:carnitine acyl transferase (CCAT)palmitoylCoA transferase I
02

Mechanism of action

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

03

Biological functions

Fatty acid metabolismTransport of long-chain fatty acidsβ-oxidationEnergy production
04

Disease associations

Deficiency disordersHypoketotic hypoglycemiaCancerMetabolic disordersHepatic encephalopathy
05

Safety considerations

Hypoketotic hypoglycemiaHepatomegalyHepatic encephalopathyNervous system damageComaSudden death
06

Biomarkers

Carnitine levelsCPT1A expression

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