AAV6-Caren

AAV6-Caren is a recombinant adeno-associated virus serotype 6 (AAV6) gene therapy vector designed to deliver the long non-coding RNA (lncRNA) *Caren* (cardiomyocyte-enriched noncoding transcript) to the heart. Developed primarily by researchers at Kumamoto University and Saga University, it is being investigated for the treatment of age-related cardiac dysfunction and heart failure. The therapeutic mechanism involves the suppression of Histidine Triad Nucleotide Binding Protein 1 (HINT1) translation. By reducing HINT1 protein levels, AAV6-Caren inhibits the Ataxia Telangiectasia Mutated (ATM)-DNA Damage Response (DDR) pathway and promotes mitochondrial biogenesis through the upregulation of Transcription Factor A, Mitochondrial (TFAM). In preclinical mouse models, intravenous administration of AAV6-Caren has been shown to improve both systolic and diastolic function and restore mitochondrial cristae in aged hearts.