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Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells
Cell-penetrating peptide-mediated delivery of phosphorodiamidate morpholino oligomers (PMOs) has shown great promise for exon-skipping therapy of Duchenne Muscular Dystrophy (DMD). Pip6a-PMO, a recently developed conjugate, is particularly efficient in a murine DMD model, although mechanisms respons...
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| Autors principals: | , , , , , , , |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
Oxford University Press
2014
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| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3950666/ https://ncbi.nlm.nih.gov/pubmed/24366877 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkt1220 |
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