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Therapeutic exon skipping for dysferlinopathies?
Antisense-mediated exon skipping is a promising therapeutic approach for Duchenne muscular dystrophy (DMD) currently tested in clinical trials. The aim is to reframe dystrophin transcripts using antisense oligonucleotides (AONs). These hide an exon from the splicing machinery to induce exon skipping...
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| 主要な著者: | , , , , , , |
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| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
Nature Publishing Group
2010
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2987387/ https://ncbi.nlm.nih.gov/pubmed/20145676 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ejhg.2010.4 |
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