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How the human protocols are structured: Onetime gene transfer dosing (not daily injections) IM injection sites distributed through target muscles Followup over months for functional endpoints Dosing Protocol (Human clinical data only) Protocol A Becker Muscular Dystrophy (Doseescalation cohorts) Low cohort: 6 10 vg/kg total (IM

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Yet this new work shows that under the extreme energetic load of hypertrophic cardiomyopathy (HCM), CK becomes the weak link : hypercontractility and mitochondrial oxidative stress inactivate CK, breaking the hearts energy relay

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Further, 34.7% achieved 25% loss, and 19.3% achieved 30% loss