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Target intelligence / Profile preview
Carnitine O-palmitoyltransferase 2 (CPT2)
- Target
- CPT2
- Molecular classification
- Enzyme, Transferase
01
Overview
Carnitine O-palmitoyltransferase 2 (CPT2) is a mitochondrial enzyme that catalyzes the conversion of acylcarnitines back into acyl-CoA esters inside mitochondria, enabling the beta-oxidation of long-chain fatty acids for energy production. It is essential for cellular energy homeostasis, and its deficiency leads to significant inherited metabolic diseases. CPT2 is a therapeutic target for metabolic disorders such as diabetes mellitus, with ongoing research focused on developing inhibitors that modulate its activity.
- Other names
- Carnitine palmitoyltransferase IIcarnitine O-palmitoyltransferase 2
02
Mechanism of action
Inhibition of CPT2 to modulate fatty acid oxidation
03
Biological functions
Fatty acid metabolismEnergy productionMitochondrial transport
04
Disease associations
Carnitine Palmitoyltransferase II deficiencyHypoketotic hypoglycemiaRhabdomyolysisLiver dysfunction
05
Safety considerations
Hypoketotic hypoglycemiaMyalgiaMuscle weakness/rhabdomyolysisLiver dysfunction/hepatomegaly
06
Biomarkers
Long-chain acylcarnitines
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