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Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice
Antisense-mediated exon skipping, which can restore the reading frame, is a most promising therapeutic approach for Duchenne muscular dystrophy. Remaining challenges include the limited applicability to patients and unclear function of truncated dystrophin proteins. Multiexon skipping targeting exon...
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| I publikationen: | Mol Ther Nucleic Acids |
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| Huvudupphovsmän: | , , , , , , , , , |
| Materialtyp: | Artigo |
| Språk: | Inglês |
| Publicerad: |
Nature Publishing Group
2015
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| Ämnen: | |
| Länkar: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4345310/ https://ncbi.nlm.nih.gov/pubmed/25647512 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2014.76 |
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