Mutation-specific dual potentiators maximize rescue of CFTR gating mutants

BACKGROUND: The potentiator ivacaftor (VX-770) has been approved for therapy of 38 cystic fibrosis (CF) mutations (~10% of the patient population) associated with a gating defect of the CF transmembrane conductance regulator (CFTR). Despite the success of VX-770 treatment of patients carrying at lea...

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Publicat a:J Cyst Fibros
Autors principals: Veit, Guido, Da Fonte, Dillon F., Avramescu, Radu G., Premchandar, Aiswarya, Bagdany, Miklos, Xu, Haijin, Bensinger, Dennis, Stubba, Daniel, Schmidt, Boris, Matouk, Elias, Lukacs, Gergely L.
Format: Artigo
Idioma:Inglês
Publicat: 2019
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7190413/
https://ncbi.nlm.nih.gov/pubmed/31678009
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jcf.2019.10.011
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