Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice

Exon-skipping therapies such as systemic i.v. administration of morpholino are being explored as a means of treating Duchenne muscular dystrophy. Here the authors show that adding a glucose-fructose mix can enhance uptake of phosphorodiamidate morpholino oligomer and its therapeutic effect in mdxmic...

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Autores principales: Gang Han, Ben Gu, Limin Cao, Xianjun Gao, Qingsong Wang, Yiqi Seow, Ning Zhang, Matthew J. A. Wood, HaiFang Yin
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2016
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Acceso en línea:https://doaj.org/article/6693a2a52ec647acbff8c2322364c6d0
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