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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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As an important hydrolytic enzyme of ceramides [25], increased Acer2 promotes the hydrolytic metabolism of ceramides, which is an important reason for the significantly decreased ceramide levels observed after hypoxic treatment