The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders.

Mol Ther Nucleic Acids · 2023 · PMC10393622 · PMID 37538053

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showed a trendno p-value reported

Although the RT-PCR result is a not an absolute quantitative measurement of exon skipping, the results clearly showed a trend of significantly enhanced exon skipping efficacy of PMO after conjugation with an EEV construct.

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