Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice.
2016
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 mdx mice.
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