Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides.

Although the in vivo data obtained did not reach statistically significant levels that might be expected to be therapeutic, the exon 4 skipping PPMO showed a clear trend towards delaying disease progression, similar to that resulting from the positive control anti-ITGA4 treatment.

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However, the data did not reach statistical significance as assessed by 2-way ANOVA analysis.

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We observed an interesting trend in exon “skippability” of the ITGA4 transcript during processing in that most in-frame exons tended to be more readily excised than out-of-frame exons.

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