Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse.

Mol Ther Methods Clin Dev · 2022 · PMC9356240 · PMID 35949298

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showed a trendno p-value reported

Overall, mice treated with ACCA neonatally at the higher dose showed a trend toward stronger dystrophin restoration at the sarcolemma compared with mice treated at 2 months of age.

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