Target intelligence / Profile preview

Long-chain 3-ketoacyl-CoA thiolase

Molecular classification
Enzyme, Thiolase
01

Overview

Long-chain 3-ketoacyl-CoA thiolase (LCKAT) is a mitochondrial enzyme that plays a critical role in the β-oxidation of long-chain fatty acids. It catalyzes the final step in this pathway, cleaving 3-ketoacyl-CoA into acetyl-CoA and a shortened acyl-CoA molecule. LCKAT is part of the mitochondrial trifunctional protein (MTP). Deficiency or mutations affecting LCKAT result in impaired oxidation of long-chain fatty acids, leading to severe metabolic disorders.

02

Mechanism of action

Inhibition of long-chain 3-ketoacyl-CoA thiolase activity

03

Biological functions

Fatty acid beta-oxidation
04

Disease associations

Fatty acid oxidation disorder (FAOD)CardiomyopathyHypoglycemiaLactic acidosis
05

Safety considerations

Impaired long-chain fatty acid oxidationNeonatal-onset cardiomyopathyRhabdomyolysisHypoglycemiaLactic acidosisEarly death if untreated
06

Interacting drugs

Trimetazidine

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