Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy.

Mol Ther · 2026 · PMC12925808 · PMID 41137396

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a positive trendno p-value reported

Conversely, the 2.1 × 10 14 vg/kg GNT0004 dose had no impact on the weight curve of WT animals, while it showed a positive trend (i.e., improvement of BW) in DMD mdx animals.

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did not reach statistical significanceno p-value reported

These differences did not reach statistical significance owing to intra-group variability.

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