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Molecular Structures Reveal Synergistic Rescue of Δ508 CFTR by Trikafta Modulators
The predominant mutation causing cystic fibrosis, the deletion of phenylalanine 508 (Δ508) in the cystic fibrosis transmembrane conductance regulator (CFTR), leads to severe defects in CFTR biogenesis and function. The advanced therapy Trikafta combines a folding corrector tezacaftor (VX-661), a cha...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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2022
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9912939/ https://www.ncbi.nlm.nih.gov/pubmed/36264792 http://dx.doi.org/10.1126/science.ade2216 |