Article
Matrine in association with FD‑2 stimulates F508del‑cystic fibrosis transmembrane conductance regulator activity in the presence of corrector VX809.
Molecular medicine reports - 1 Dec 2017
Marengo Barbara, Speciale Andrea, Senatore Lisa, Garibaldi Silvano, Musumeci Francesca, Nieddu Erika, Pollarolo Benedetta, Pronzato Maria Adelaide, Schenone Silvia, Mazzei Mauro, Domenicotti Cinzia
Abstract excerpt
Cystic fibrosis is caused by mutations of the cystic fibrosis transmembrane conductance regulator (CFTR) gene, and the predominant mutation is termed Phe508del (F508del). Therapy for F508del‑CFTR patients is based on the use of Orkambi®, a combination of VX809 and VX770. However, though Orkambi leads to an improvement in the lung function of patients, a progressive reduction in its efficacy has been observed. In...
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