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Schematic of the experimental design depicting AAV9 delivery and subsequent evaluation of disease. Dual-AAV9 ABE8e or single AAV9-Cas9 were injected (solid filled arrow, first dose; unfilled arrow, second dose) into R403Q-129SvEv or R403Q-129SvEv/S4 mice at postnatal weeks 2-3. All R403Q-129SvEv/S4 mice consistently develop LVH by 8-10 weeks (gray box), whereas only male R403Q-129SvEv mice show LVH at 20-25 weeks (blue box). Cardiac morphology and function were assessed by echocardiography at 2-4-week intervals.

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