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In Vitro Multiexon Skipping by Antisense PMOs in Dystrophic Dog and Exon 7-Deleted DMD Patient

Antisense oligonucleotide induced exon skipping emerges as a promising therapeutic strategy for patients suffering from a devastating muscle disorder Duchenne muscular dystrophy (DMD). Systemic administration of antisense phosphorodiamidate morpholino oligomers (PMOs) targeting exons 6 and 8 in dyst...

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Detalles Bibliográficos
Publicado en:Methods Mol Biol
Autores principales: Nakamura, Akinori, Aoki, Yoshitsugu, Tsoumpra, Maria, Yokota, Toshifumi, Takeda, Shin’ichi
Formato: Artigo
Lenguaje:Inglês
Publicado: 2018
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC6557157/
https://ncbi.nlm.nih.gov/pubmed/30171540
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/978-1-4939-8651-4_9
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