What question did this study set out to answer?

To evaluate the effectiveness of systemic antisense oligonucleotide delivery for skipping exons 45-55 in dystrophic mdx52 mice.

Bodywide skipping of exons 45–55 in dystrophic mdx52 mice by systemic antisense delivery

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Randomized trial shows effective skipping of exons 45-55 in dystrophic mice, suggesting therapeutic potential for muscular dystrophy.

To evaluate the effectiveness of systemic antisense oligonucleotide delivery for skipping exons 45-55 in dystrophic mdx52 mice.
Designed morpholino mixtures to minimize duplex formation
Tested in H2K-mdx52 myotubes and mdx52 mice
Performed systemic injections of 10 AOs to assess dystrophin expression
Systemic injection of 10 AOs led to up to 15% wild-type dystrophin protein levels in skeletal muscles
Improved muscle strength and histopathology were observed
No detectable toxicity in treated mdx52 mice

Aoki et al. (2012) studied this question.

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