A short antisense oligonucleotide ameliorates symptoms of severe mouse models of spinal muscular atrophy.

Mol Ther Nucleic Acids · 2014 · PMC4121513 · PMID 25004100

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did not reach statistical significanceno p-value reported

36 Treatment with 3UP8i resulted in a corrective, downward trend toward heterozygote levels, but it did not reach statistical significance compared to CON8i-treated SMA mice by P18.

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an encouraging trendno p-value reported

A high dose (80 μg/g body weight) of 3UP8i, we observed an encouraging trend of body weight gain, improved righting reflex, and a modest 3-day extension in survival of these severe mice.

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