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Target intelligence / Profile preview
Carnitine-acylcarnitine translocase (CACT)
- Target
- CACT
- Molecular classification
- Transporter, Mitochondrial inner membrane protein, Solute Carrier
01
Overview
Carnitine-acylcarnitine translocase (CACT) is a mitochondrial inner membrane protein essential for the transport of long-chain fatty acylcarnitines into the mitochondrial matrix, where they undergo β-oxidation to produce energy. CACT operates as part of the carnitine shuttle system, exchanging acylcarnitines from the intermembrane space with free carnitine from the matrix in a 1:1 ratio. Deficiency or dysfunction of CACT leads to Carnitine-Acylcarnitine Translocase Deficiency (CACT deficiency), an autosomal recessive disorder caused by mutations in SLC25A20.
- Other names
- CACCarnitine-acylcarnitine carriersolute carrier family 25 member 20
02
Mechanism of action
L-carnitine supplementation supports residual CACT function and helps in the excretion of acylcarnitines.
03
Biological functions
Transport of short-, medium-, and long-chain acyl-carnitines across the inner mitochondrial membraneExchange of acylcarnitines from the intermembrane space with free carnitine from the matrixFacilitates β-oxidation of fatty acids
04
Disease associations
Carnitine-Acylcarnitine Translocase Deficiency (CACT deficiency)Disorder of fatty acid metabolism
05
Safety considerations
Metabolic crises triggered by fasting or illnessSevere hypoketotic hypoglycemiaHyperammonemiaCardiomyopathy/arrhythmiasHepatic dysfunction/liver damageSkeletal muscle weakness/myopathy/rhabdomyolysisEncephalopathy/developmental delay/seizures
06
Interacting drugs
L-carnitine
07
Biomarkers
Acylcarnitine levelsSLC25A20 mutations
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