Long-term Exon Skipping Studies With 2′-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models

Antisense-mediated exon skipping for Duchenne muscular dystrophy (DMD) is currently tested in phase 3 clinical trials. The aim of this approach is to modulate splicing by skipping a specific exon to reframe disrupted dystrophin transcripts, allowing the synthesis of a partly functional dystrophin pr...

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Hlavní autoři: Tanganyika-de Winter, Christa L, Heemskerk, Hans, Karnaoukh, Tatyana G, van Putten, Maaike, de Kimpe, Sjef J, van Deutekom, Judith, Aartsma-Rus, Annemieke
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2012
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3464881/
https://ncbi.nlm.nih.gov/pubmed/23344236
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2012.38
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