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Skipping Multiple Exons to Treat DMD—Promises and Challenges
Duchenne muscular dystrophy (DMD) is a lethal disorder caused by mutations in the DMD gene. Antisense-mediated exon-skipping is a promising therapeutic strategy that makes use of synthetic nucleic acids to skip frame-disrupting exon(s) and allows for short but functional protein expression by restor...
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| 出版年: | Biomedicines |
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| 主要な著者: | , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
MDPI
2018
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5874658/ https://ncbi.nlm.nih.gov/pubmed/29301272 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/biomedicines6010001 |
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