Mechanism-based corrector combination restores ΔF508-CFTR folding and function

The most common cystic fibrosis (CF) mutation, ΔF508 in the nucleotide binding domain-1 (NBD1), impairs CFTR coupled-domain folding, plasma membrane (PM) expression, function and stability. VX-809, a promising investigational corrector of ΔF508-CFTR misprocessing, has limited clinical benefit and in...

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Detalhes bibliográficos
Main Authors: Okiyoneda, Tsukasa, Veit, Guido, Dekkers, Johanna F., Bagdany, Miklos, Soya, Naoto, Xu, Haijin, Roldan, Ariel, Verkman, Alan S., Kurth, Mark, Simon, Agnes, Hegedus, Tamas, Beekman, Jeffrey M., Lukacs, Gergely L.
Formato: Artigo
Idioma:Inglês
Publicado em: 2013
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3840170/
https://ncbi.nlm.nih.gov/pubmed/23666117
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/nchembio.1253
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