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Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression.
Veit G, Avramescu RG, Perdomo D, Phuan PW, Bagdany M, Apaja PM, Borot F, Szollosi D, Wu YS, Finkbeiner WE, Hegedus T, Verkman AS, Lukacs GL. Veit G, et al. Among authors: finkbeiner we. Sci Transl Med. 2014 Jul 23;6(246):246ra97. doi: 10.1126/scitranslmed.3008889. Sci Transl Med. 2014. PMID: 25101887 Free PMC article.
Inhibitors of pendrin anion exchange identified in a small molecule screen increase airway surface liquid volume in cystic fibrosis.
Haggie PM, Phuan PW, Tan JA, Zlock L, Finkbeiner WE, Verkman AS. Haggie PM, et al. Among authors: finkbeiner we. FASEB J. 2016 Jun;30(6):2187-97. doi: 10.1096/fj.201600223R. Epub 2016 Mar 1. FASEB J. 2016. PMID: 26932931 Free PMC article.
Studies using pendrin knockout mice and airway epithelial cells from hearing-impaired subjects with pendrin loss of function suggest involvement of pendrin in inflammatory lung diseases, including cystic fibrosis (CF), perhaps by regulation of airway surface liquid (ASL) volume. …
Studies using pendrin knockout mice and airway epithelial cells from hearing-impaired subjects with pendrin loss of function suggest involve …
CFTR modulator therapy for cystic fibrosis caused by the rare c.3700A>G mutation.
Phuan PW, Haggie PM, Tan JA, Rivera AA, Finkbeiner WE, Nielson DW, Thomas MM, Janahi IA, Verkman AS. Phuan PW, et al. Among authors: finkbeiner we. J Cyst Fibros. 2021 May;20(3):452-459. doi: 10.1016/j.jcf.2020.07.003. Epub 2020 Jul 14. J Cyst Fibros. 2021. PMID: 32674984 Free PMC article.
151 results