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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.
Combination potentiator ('co-potentiator') therapy for CF caused by CFTR mutants, including N1303K, that are poorly responsive to single potentiators.
Phuan PW, Son JH, Tan JA, Li C, Musante I, Zlock L, Nielson DW, Finkbeiner WE, Kurth MJ, Galietta LJ, Haggie PM, Verkman AS. Phuan PW, et al. Among authors: finkbeiner we. J Cyst Fibros. 2018 Sep;17(5):595-606. doi: 10.1016/j.jcf.2018.05.010. Epub 2018 Jun 12. J Cyst Fibros. 2018. PMID: 29903467 Free PMC article.
151 results