Single-AAV-mediated cardiomyocyte-specific adenine base editing ameliorates cardiac hypertrophy in vivo
Hypertrophic cardiomyopathy (HCM) is the most common inherited cardiac disorder, frequently accompanied by cardiac hypercontractility, diastolic dysfunction, heart failure and sudden cardiac death. The disease is predominantly caused by pathogenic variants in genes encoding sarcomeric proteins and current therapeutic strategies largely focus on symptom management. Adeno-associated virus-based…
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