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Rescue of multiple class II CFTR mutations by elexacaftor+tezacaftor+ivacaftor mediated in part by the dual activities of elexacaftor as both corrector and potentiator
Positive results in pre-clinical studies of the triple combination of elexacaftor, tezacaftor and ivacaftor, performed in airway epithelial cell cultures obtained from patients harbouring the class II cystic fibrosis transmembrane conductance regulator (CFTR) mutation F508del-CFTR, translated to imp...
Autores principales: | Laselva, Onofrio, Bartlett, Claire, Gunawardena, Tarini N.A., Ouyang, Hong, Eckford, Paul D.W., Moraes, Theo J., Bear, Christine E., Gonska, Tanja |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
European Respiratory Society
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8209484/ https://www.ncbi.nlm.nih.gov/pubmed/33303536 http://dx.doi.org/10.1183/13993003.02774-2020 |
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