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Rational Design of Antisense Oligomers to Induce Dystrophin Exon Skipping
Duchenne muscular dystrophy (DMD), one of the most severe neuromuscular disorders of childhood, is caused by the absence of a functional dystrophin. Antisense oligomer (AO) induced exon skipping is being investigated to restore functional dystrophin expression in models of muscular dystrophy and DMD...
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| Autors principals: | , , , , , |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
Nature Publishing Group
2009
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2835229/ https://ncbi.nlm.nih.gov/pubmed/19293776 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.49 |
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