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Personalized exon skipping strategies to address clustered non-deletion dystrophin mutations
Antisense oligomer induced exon skipping is showing promise as a therapy to reduce the severity of Duchenne Muscular Dystrophy. To date, the focus has been on excluding single exons flanking frame-shifting deletions in the dystrophin gene, however, a third of all Duchenne Muscular Dystrophy causing...
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| Main Authors: | , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2010
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2991584/ https://ncbi.nlm.nih.gov/pubmed/20817455 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.nmd.2010.07.276 |
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