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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

44-46 Unlike the other vitreous opacities described here, asteroid hyalosis has little effect on vision and affected patients are generally asymptomatic

L-carnitine is a naturally occurring amino acid that helps your body generate energy from fat

CoQH 2 is a free radical-trapping antioxidant that prevents lipid peroxidation and inhibits ferroptosis

Sulhan et al., 2020), leading to a sharp increase in intracranial pressure and eventually cerebral hernia (Winkler et al., 2016)