Clinically translatable mitochondrial gene therapy in muscle using tandem mtZFN architecture.

Despite this, a clear trend in mtCN change could be observed, particularly in the dose-dependent AAVMYO experiment, where the trend of mtCN is negatively correlated with dose, with the highest dose nearing significance ( p = 0.067) (Fig. 7D ).

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Despite this, a clear trend in mtCN change could be observed, particularly in the dose-dependent AAVMYO experiment, where the trend of mtCN is negatively correlated with dose, with the highest dose nearing significance ( p = 0.067) (Fig. 7D ).

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Highly significant reduction in heteroplasmy in the right legs versus the control left legs of both treatment groups was detected, with separate capsid delivery, achieving a ~28% reduction in mutation load, while nearly 40% reduction observed for the tandem administration (Fig. 4B ).

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