Antisense PMO Found in Dystrophic Dog Model Was Effective in Cells from Exon 7-Deleted DMD Patient

BACKGROUND: Antisense oligonucleotide-induced exon skipping is a promising approach for treatment of Duchenne muscular dystrophy (DMD). We have systemically administered an antisense phosphorodiamidate morpholino oligomer (PMO) targeting dystrophin exons 6 and 8 to a dog with canine X-linked muscula...

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Detalhes bibliográficos
Main Authors: Saito, Takashi, Nakamura, Akinori, Aoki, Yoshitsugu, Yokota, Toshifumi, Okada, Takashi, Osawa, Makiko, Takeda, Shin'ichi
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2010
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Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2923599/
https://ncbi.nlm.nih.gov/pubmed/20805873
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0012239
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