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Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs
OBJECTIVE: Duchenne muscular dystrophy (DMD) is caused by the inability to produce dystrophin protein at the myofiber membrane. A method to rescue dystrophin production by antisense oligonucleotides, termed `exon-skipping', has been reported for the mdx mouse and in four DMD patients by local i...
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| Udgivet i: | Ann Neurol |
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| Main Authors: | , , , , , , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
2009
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5951302/ https://ncbi.nlm.nih.gov/pubmed/19288467 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.21627 |
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