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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| Main Authors: | , , , , , , , , |
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| Format: | article |
| Language: | EN |
| Published: |
Nature Portfolio
2016
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| Subjects: | |
| Online Access: | https://doaj.org/article/6693a2a52ec647acbff8c2322364c6d0 |
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| Summary: | 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 mdxmice. |
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