Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice

Antisense-mediated exon skipping, which can restore the reading frame, is a most promising therapeutic approach for Duchenne muscular dystrophy. Remaining challenges include the limited applicability to patients and unclear function of truncated dystrophin proteins. Multiexon skipping targeting exon...

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Publicat a:Mol Ther Nucleic Acids
Autors principals: Echigoya, Yusuke, Aoki, Yoshitsugu, Miskew, Bailey, Panesar, Dharminder, Touznik, Aleksander, Nagata, Tetsuya, Tanihata, Jun, Nakamura, Akinori, Nagaraju, Kanneboyina, Yokota, Toshifumi
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2015
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4345310/
https://ncbi.nlm.nih.gov/pubmed/25647512
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2014.76
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