Gosset Molecular Classification
Enzyme, Transferase
Other Names
Carnitine palmitoyltransferase II, carnitine O-palmitoyltransferase 2
Disease Roles
Carnitine O-palmitoyltransferase 2 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.
Mechanism of Action
Inhibition of CPT2 to modulate fatty acid oxidation
Biological Functions
Fatty acid metabolism
Energy production
Mitochondrial transport
Disease Associations
Carnitine Palmitoyltransferase II deficiency
Hypoketotic hypoglycemia
Rhabdomyolysis
Liver dysfunction
Safety Considerations
- Hypoketotic hypoglycemia
- Myalgia
- Muscle weakness/rhabdomyolysis
- Liver dysfunction/hepatomegaly
Associated Biomarkers
| Biomarker |
|---|
| Long-chain acylcarnitines |