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Structure-guided combination therapy to potently improve the function of mutant CFTRs
Available drugs are unable to effectively rescue the folding defects in vitro and ameliorate the clinical-phenotype of cystic fibrosis (CF), caused by deletion of F508 (ΔF508 or F508del) and some point mutations in the CF transmembrane conductance regulator (CFTR), a plasma membrane (PM) anion chann...
Autores principales: | Veit, Guido, Xu, Haijin, Dreano, Elise, Avramescu, Radu G, Bagdany, Miklos, Beitel, Lenore K, Roldan, Ariel, Hancock, Mark A, Lay, Cecilia, Li, Wei, Morin, Katelin, Gao, Sandra, Mak, Puiying A, Ainscow, Edward, Orth, Anthony P, McNamara, Peter, Edelman, Aleksander, Frenkiel, Saul, Matouk, Elias, Sermet-Gaudelus, Isabelle, Barnes, William G, Lukacs, Gergely L |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6301090/ https://www.ncbi.nlm.nih.gov/pubmed/30297908 http://dx.doi.org/10.1038/s41591-018-0200-x |
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