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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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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: Yin, HaiFang, Moulton, Hong M, Betts, Corinne, Merritt, Thomas, Seow, Yiqi, Ashraf, Shirin, Wang, QingSong, Boutilier, Jordan, Wood, Matthew JA
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Nature Publishing Group 2010
Pynciau:
Mynediad Ar-lein: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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