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AAV-CARF
- Development stage
- Preclinical
- Lead developer
- Charles Drew University of Medicine and Science
- Modality
- Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
- Administration
- Intravenous
01
Overview
AAV-CARF is an adeno-associated virus (AAV) vector-based gene therapy designed to overexpress the CDKN2A-interacting protein (CARF), also known as CDKN2AIP, in hepatocytes. CARF plays a critical role in regulating mitochondrial fatty acid oxidation (FAO) by activating the FAO/PPAR pathway, including key enzymes such as CPT2 and ACADM. In metabolic dysfunction-associated steatotic liver disease (MASLD) and obesity, CARF expression is often reduced, contributing to impaired lipid metabolism and steatosis. By restoring CARF levels, AAV-CARF promotes mitochondrial oxidative metabolism, reduces hepatic triglyceride accumulation, and improves the metabolic profile of the liver. This therapeutic approach is currently being investigated in preclinical models for its potential to treat obesity-associated hepatic steatosis.
- Other names
- adeno-associated virus-CARFAAV-CDKN2AIPAAV-CDKN-2AIPAAV-CDKN 2AIP
02
Targets
CDKN2AIP (CDKN2A interacting protein)
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