Antisense oligonucleotides targeting valosin-containing protein ameliorate muscle pathology and molecular defects in cell and mouse models of multisystem proteinopathy.

Clin Transl Med · 2025 · PMC12683293 · PMID 41355735

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did not reach statistical significanceno p-value reported

Motor testing data trended for improved performance in the inverted screen test with VCP ASO treatment in A232E mice but did not reach statistical significance (Figure 6D ).

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