Single-AAV-mediated cardiomyocyte-specific adenine base editing ameliorates cardiac hypertrophy in vivo
Thomas Monnikhof, Su Ji Han, Laura Stumpo…·Eva van Rooij
This paper develops a single-AAV vector for cardiomyocyte-specific adenine base editing to correct a mutation causing HCM in mice, achieving therapeutic effects at lower doses than dual-AAV approaches.