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Splicing modulation therapy in the treatment of genetic diseases
Antisense-mediated splicing modulation is a tool that can be exploited in several ways to provide a potential therapy for rare genetic diseases. This approach is currently being tested in clinical trials for Duchenne muscular dystrophy and spinal muscular atrophy. The present review outlines the ver...
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| 出版年: | Appl Clin Genet |
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| 主要な著者: | , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
Dove Medical Press
2014
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4259397/ https://ncbi.nlm.nih.gov/pubmed/25506237 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/TACG.S71506 |
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