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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Hlavní autoři: Yin, HaiFang, Moulton, Hong M, Betts, Corinne, Merritt, Thomas, Seow, Yiqi, Ashraf, Shirin, Wang, QingSong, Boutilier, Jordan, Wood, Matthew JA
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2010
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Original Article
On-line přístup: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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