Exon skipping peptide-conjugated morpholinos downregulate dynamin 2 to rescue centronuclear myopathy.

Brain · 2025 · PMC12677909 · PMID 40608957

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a clear trendno p-value reported

normally spliced) Dnm2 mRNA levels, a clear trend for total Dnm2 mRNA downregulation and a 2.8-fold decrease in DNM2 protein levels, indicating a promising compound ( Fig. 1B and C and Supplementary Fig. 4B ).

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