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Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or two F508del alleles

Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator gene (CFTR) that result in diminished quantity and/or function of the CFTR anion channel. F508del-CFTR, the most common CF-causing mutation (found in ∼90% of patients), causes severe processing and trafficking...

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Xehetasun bibliografikoak
Egile Nagusiak: Jennifer L. Taylor-Cousar, Marcus A. Mall, Bonnie W. Ramsey, Edward F. McKone, Elizabeth Tullis, Gautham Marigowda, Charlotte M. McKee, David Waltz, Samuel M. Moskowitz, Jessica Savage, Fengjuan Xuan, Steven M. Rowe
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: European Respiratory Society 2019-06-01
Saila:ERJ Open Research
Sarrera elektronikoa:http://openres.ersjournals.com/content/5/2/00082-2019.full
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