Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia

  title={Phenotypic profiling of CFTR modulators in patient-derived respiratory epithelia},
  author={Saumel Ahmadi and Zolt{\'a}n Boz{\'o}ky and Michelle Di Paola and Sunny Xia and Canhui Li and Amy P Wong and Leigh Wellhauser and Steven V Molinski and Wan F. Ip and Hong Ouyang and Julie Avolio and Julie D. Forman-Kay and Felix Ratjen and Jeremy A. Hirota and Johanna M Rommens and Janet Rossant and Tanja Gonska and Theo J Moraes and Christine E Bear},
  booktitle={npj Genomic Medicine},
Pulmonary disease is the major cause of morbidity and mortality in patients with cystic fibrosis, a disease caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene. Heterogeneity in CFTR genotype–phenotype relationships in affected individuals plus the escalation of drug discovery targeting specific mutations highlights the need to develop robust in vitro platforms with which to stratify therapeutic options using relevant tissue. Toward this goal, we adapted a… CONTINUE READING

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