期刊
RNA BIOLOGY
卷 7, 期 4, 页码 453-461出版社
TAYLOR & FRANCIS INC
DOI: 10.4161/rna.7.4.12264
关键词
antisense oligonucleotides; exon skipping; neuromuscular disorders; therapy; splicing; clinical trials
资金
- Duchenne Parent Project (the Netherlands)
- ZonMW (the Netherlands)
- NWO (the Netherlands)
- Prinses Beatrix Fonds (the Netherlands)
- EU [LSHM-CT-2006-036825, HEALTH-F2-2009-241665]
While disruption of alternative splicing underlies many diseases, modulation of splicing using antisense oligonucleotides (AONs) can have therapeutic implications. The most notable example is Duchenne muscular dystrophy (DMD), where antisense-mediated exon skipping can restore the open reading frame and allow the synthesis of partly functional dystrophin proteins instead of non-functional ones. This approach is currently tested in early phase clinical trials. In this review the development of the exon skipping approach in patient-derived cell cultures, animal models and patients is described and hurdles that have to be overcome to make this personalized medicine type approach widely applicable are discussed.
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