Cas13-Mediated RNA Base Editing for the Treatment of Hereditary Hypertrophic Cardiomyopathy

we created a humanized HCM mouse model carrying the MYH7 R403Q pathogenic mutation, which exhibited significant ventricular hypertrophy, myocyte disarray, and fibrosis. We then utilized RNA base editor (mxABE) to convert the CAG (glutamine) to CGG (arginine) in the mutant RNA, effectively restoring cardiac function in the HCM mice after systemic administration, which demonstrate the potential of mxABE to treat inherited cardiac diseases.