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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Bibliografiska uppgifter
I publikationen:Mol Ther Nucleic Acids
Huvudupphovsmän: Echigoya, Yusuke, Aoki, Yoshitsugu, Miskew, Bailey, Panesar, Dharminder, Touznik, Aleksander, Nagata, Tetsuya, Tanihata, Jun, Nakamura, Akinori, Nagaraju, Kanneboyina, Yokota, Toshifumi
Materialtyp: Artigo
Språk:Inglês
Publicerad: Nature Publishing Group 2015
Ämnen:
Original Article
Länkar: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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