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Activity of lumacaftor is not conserved in zebrafish Cftr bearing the major cystic fibrosis‐causing mutation

Abstract F508del‐cystic fibrosis transmembrane conductance regulator (CFTR) is the major mutant responsible for cystic fibrosis (CF). ORKAMBI®, approved for patients bearing this mutant, contains lumacaftor (VX‐809) that partially corrects F508del‐CFTR's processing defect and ivacaftor (VX‐770) that...

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Bibliografiset tiedot
Päätekijät: Onofrio Laselva, Steven Erwood, Kai Du, Zhenya Ivakine, Christine E. Bear
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Wiley 2019-10-01
Sarja:FASEB BioAdvances
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Linkit:https://doi.org/10.1096/fba.2019-00039
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