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 |
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Formato: | article |
Lenguaje: | EN |
Publicado: |
Nature Portfolio
2016
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Materias: | |
Acceso en línea: | https://doaj.org/article/6693a2a52ec647acbff8c2322364c6d0 |
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