CPT1a gene therapy

CPT1a gene therapy is an experimental adeno-associated virus (AAV)-based gene therapy designed to overexpress carnitine palmitoyltransferase 1A (CPT1a), the rate-limiting enzyme for long-chain fatty acid oxidation in mitochondria. Developed primarily in academic research settings, notably at The Ohio State University, the therapy utilizes AAV9 vectors to deliver either the wild-type CPT1a gene or a constitutively active mutant form (CPT1AM). In the context of heart failure, cardiac-specific delivery (often utilizing the cTnT promoter) aims to rescue contractile function and suppress adverse remodeling by restoring fatty acid metabolism and modulating non-canonical signaling pathways, such as histone acetylation. In metabolic contexts, liver-directed delivery is investigated to reduce hepatic steatosis and improve glucose homeostasis in models of obesity and nonalcoholic fatty liver disease (NAFLD).

Other names

AAV-CPT1aAAV-CPT-1aAAV-CPT 1aCPT1AM gene therapyCPT-1AM gene therapyCPT 1AM gene therapyAAV9-CPT1aAAV-9-CPT1aAAV 9-CPT1a