Carnitine palmitoyltransferase 1 (CPT1) (CPT1)

Carnitine palmitoyltransferase 1 (CPT1) is a critical regulatory enzyme located on the outer mitochondrial membrane that facilitates the transport of long-chain fatty acids into the mitochondria for beta-oxidation (UniProt P08559, Q92523). It catalyzes the conversion of long-chain acyl-CoAs into acylcarnitines, which is the rate-limiting step in fatty acid catabolism (PubMed: 9174460). The enzyme exists in three primary isoforms: CPT1A (liver), CPT1B (muscle and heart), and CPT1C (brain), each with distinct regulatory properties and tissue distribution (PubMed: 15833718). In metabolic disorders such as type 2 diabetes, CPT1 inhibition is explored as a strategy to reduce hepatic gluconeogenesis and improve glucose utilization by shifting the cellular energy balance (PubMed: 21839406). Pharmacological agents like Etomoxir and Perhexiline have been studied as CPT1 inhibitors, though their clinical use is often limited by safety concerns such as hepatotoxicity and potential cardiac adverse effects (DrugBank DB01074). Modern drug discovery efforts focus on developing isoform-specific or reversible inhibitors to mitigate these risks while maintaining therapeutic efficacy in treating metabolic and cardiovascular diseases (PubMed: 25651180).

Other names

CPT ICarnitine O-palmitoyltransferase 1CPT1ACPT1BL-CPT1M-CPT1

Inhibition of the conversion of long-chain fatty acyl-CoA to acylcarnitine at the outer mitochondrial membrane, thereby blocking the entry of fatty acids into the mitochondria for beta-oxidation.