Emerging preclinical modulators developed for F508del-CFTR have the potential to be effective for ORKAMBI resistant processing mutants

Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del<i>CFTR</i>

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Medicine, Pharmacology, Pharmacotherapy +15

Processing of mutant cystic fibrosis transmembrane conductance regulator is temperature-sensitive

Gerene M. Denning, Matthew P. Anderson, Jane F. Amara et al. · Nature · 1992 · 1.2K citations

Life Sciences, Genetics, Molecular Biology +9

Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

Fredrick Van Goor, Sabine Hadidaꝉ, Peter D. J. Grootenhuis et al. · Proceedings of the National Academy of Sciences · 2009 · 1.2K citations · Full text

Medicine, Cell Biology, Pharmacology +17

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809

Fredrick Van Goor, Sabine Hadidaꝉ, Peter D. J. Grootenhuis et al. · Proceedings of the National Academy of Sciences · 2011 · 1.1K citations · Full text

Medicine, Molecular Biology, Pharmacology +13

CFTR Expression and Chloride Secretion in Polarized Immortal Human Bronchial Epithelial Cells

Alison Cozens, M J Yezzi, Karl Kunzelmann et al. · American Journal of Respiratory Cell and Molecular Biology · 1994 · 935 citations

Life Sciences, Cell Biology, Molecular Biology +16