Article
Discovery of GLPG2737, a Potent Type 2 Corrector of CFTR for the Treatment of Cystic Fibrosis in Combination with a Potentiator and a Type 1 Co-corrector.
Journal of medicinal chemistry - 11 Apr 2024
Pizzonero Mathieu, Akkari Rhalid, Bock Xavier, Gosmini Romain, De Lemos Elsa, Duthion Béranger, Newsome Gregory, Mai Thi-Thu-Trang, Roques Virginie, Jary Hélène, Lefrancois Jean-Michel, Cherel Laetitia, Quenehen Vanessa, Babel Marielle, Merayo Nuria, Bienvenu Natacha, Mammoliti Oscar, Coti Ghjuvanni, Palisse Adeline, Cowart Marlon, Shrestha Anurupa, Greszler Stephen, Van Der Plas Steven, Jansen Koen, Claes Pieter, Jans Mia, Gees Maarten, Borgonovi Monica, De Wilde Gert, Conrath Katja
Abstract excerpt
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR) protein. This epithelial anion channel regulates the active transport of chloride and bicarbonate ions across membranes. Mutations result in reduced surface expression of CFTR channels with impaired functionality. Correctors are small molecules that support the trafficking of CFTR to increase its membrane expression....
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