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Muscular dystrophy

New exon-skipping strategy rescues dystrophin

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From Nature Reviews Drug Discovery

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References

  1. Goyenvalle, A. et al. Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers. Nature Med. http://dx.doi.org/10.1038/nm.3765 (2015)

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Crunkhorn, S. New exon-skipping strategy rescues dystrophin. Nat Rev Drug Discov 14, 236–237 (2015). https://doi.org/10.1038/nrd4587

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  • DOI: https://doi.org/10.1038/nrd4587

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