Effective Dystrophin Restoration by a Novel Muscle-Homing Peptide–Morpholino Conjugate in Dystrophin-Deficient mdx Mice

Antisense oligonucleotide (AO)–mediated splice correction therapy for Duchenne muscular dystrophy has shown huge promise from recent phase 2b clinical trials, however high doses and costs are required and targeted delivery can lower both of these. We have previously demonstrated the feasibility of t...

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Главные авторы: Gao, Xianjun, Zhao, Jingwen, Han, Gang, Zhang, Yajie, Dong, Xue, Cao, Limin, Wang, Qingsong, Moulton, Hong M, Yin, HaiFang
Формат: Artigo
Язык:Inglês
Опубликовано: Nature Publishing Group 2014
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Original Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC4088999/
https://ncbi.nlm.nih.gov/pubmed/24732757
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.63
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