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Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice - Nature Medicine

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Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice - Nature Medicine

Dominant missense pathogenic variants in cardiac myosin heavy chain cause hypertrophic cardiomyopathy (HCM), a currently incurable disorder that increases risk for stroke, heart failure and sudden cardiac death. In this study, we assessed two different genetic therapies—an adenine base editor (ABE8e) and a potent Cas9 nuclease delivered by AAV9—to prevent disease in mice carrying the heterozygous HCM pathogenic variant myosin R403Q.

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Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice - Nature Medicine

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Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice - Nature Medicine

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