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Novel Correctors and Potentiators Enhance Translational Readthrough in CFTR Nonsense Mutations
Premature-termination codons (PTCs) in CFTR (cystic fibrosis [CF] transmembrane conductance regulator) result in nonfunctional CFTR protein and are the proximate cause of ∼11% of CF-causing alleles, for which no treatments exist. The CFTR corrector lumacaftor and the potentiator ivacaftor improve CF...
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| Опубликовано в: : | Am J Respir Cell Mol Biol |
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| Главные авторы: | , , , , , , , , , |
| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
American Thoracic Society
2021
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| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8086047/ https://ncbi.nlm.nih.gov/pubmed/33616476 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1165/rcmb.2019-0291OC |
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