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Nanomolar-potency ‘co-potentiator’ therapy for cystic fibrosis caused by a defined subset of minimal function CFTR mutants

Available CFTR modulators provide no therapeutic benefit for cystic fibrosis (CF) caused by many loss-of-function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel, including N1303K. We previously introduced the concept of ‘co-potentiators’ (combination-pot...

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Detalles Bibliográficos
Autores principales:	Phuan, Puay-Wah, Tan, Joseph-Anthony, Rivera, Amber A., Zlock, Lorna, Nielson, Dennis W., Finkbeiner, Walter E., Haggie, Peter M., Verkman, Alan S.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2019
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6881293/ https://www.ncbi.nlm.nih.gov/pubmed/31776420 http://dx.doi.org/10.1038/s41598-019-54158-2

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