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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Autores principales: Gao, Xianjun, Zhao, Jingwen, Han, Gang, Zhang, Yajie, Dong, Xue, Cao, Limin, Wang, Qingsong, Moulton, Hong M, Yin, HaiFang
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2014
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Original Article
Acceso en línea: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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