AOC 1044 induces exon 44 skipping and restores dystrophin protein in preclinical models of Duchenne muscular dystrophy.

Nucleic Acids Res · 2025 · PMC11969676 · PMID 40183632

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did not reach statistical significanceno p-value reported

1C ), indicating that exon 44 skipping resulted in the formation of mature DMD mRNA, although the increase did not reach statistical significance.

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