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A novel triple combination of pharmacological chaperones improves F508del-CFTR correction
Pharmacological chaperones (e.g. VX-809, lumacaftor) that bind directly to F508del-CFTR and correct its mislocalization are promising therapeutics for Cystic Fibrosis (CF). However to date, individual correctors provide only ~4% improvement in lung function measured as FEV1, suggesting that multiple...
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| 出版年: | Sci Rep |
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| 主要な著者: | , , , , , , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
Nature Publishing Group UK
2018
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6065411/ https://ncbi.nlm.nih.gov/pubmed/30061653 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-018-29276-y |
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