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The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is associated with mutations in the dystrophin gene that disrupt the open reading frame whereas the milder Becker's form is associated with mutations which leave an in-frame mRNA transcript that can be translated into a protein that includes the N- and C- termi...
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| Main Authors: | , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado: |
Nature Publishing Group
2011
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| Assuntos: | |
| Acceso en liña: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3017449/ https://ncbi.nlm.nih.gov/pubmed/20978473 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.219 |
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