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Smith SR, Aronne LJ, Burns CM, Kesty NC, Halseth AE, Weyer C

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Gordon, R

The LEADER trial was an international, randomised, double-blind, controlled cardiovascular outcomes trial (CVOT)

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90,91 However, gene editing approaches remain effective in treating inherent mtDNA mutations, while the ROS-induced sporadic and clonally expanding mtDNA mutations observed in -thalassemia and related hemoglobinopathies present challenges for current gene editing therapeutic strategies