Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO

Splice modulation using antisense oligonucleotides (AOs) has been shown to yield targeted exon exclusion to restore the open reading frame and generate truncated but partially functional dystrophin protein. This has been successfully demonstrated in dystrophin-deficient mdx mice and in Duchenne musc...

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Bibliografiska uppgifter
Huvudupphovsmän: Yin, HaiFang, Moulton, Hong M, Betts, Corinne, Merritt, Thomas, Seow, Yiqi, Ashraf, Shirin, Wang, QingSong, Boutilier, Jordan, Wood, Matthew JA
Materialtyp: Artigo
Språk:Inglês
Publicerad: Nature Publishing Group 2010
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Original Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC2951563/
https://ncbi.nlm.nih.gov/pubmed/20700113
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.151
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