Using a State-of-the-Art Toolbox to Evaluate Molecular and Functional Readouts of Antisense Oligonucleotide-Induced Exon Skipping in <i>mdx</i> Mice.

The corresponding gait discriminant score displayed a highly significant ( P < 0.0001) differentiation between the mdx mice and WT controls and a significant shift toward WT in the SC and IV M23D treatment groups compared to the scrambled control group ( P < 0.05), indicating an M23D treatment effect on overall motor performance ( Fig. 7B ).

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The difference between mdx mice treated with scrambled (SCR) M23D and WT controls was significant for PCs 1 and 2 and displayed a trend toward significance ( P = 0.07) for PC3.

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