Mathieu Pizzonero, Rhalid Akkari, Xavier Bock, Romain Gosmini, Elsa De Lemos, Béranger Duthion, Gregory Newsome, Thi-Thu-Trang Mai, Virginie Roques, Hélène Jary, Jean-Michel Lefrancois, Laetitia Cherel, Vanessa Quenehen, Marielle Babel, Nuria Merayo, Natacha Bienvenu, Oscar Mammoliti, Ghjuvanni Coti, Adeline Palisse, Marlon Cowart, Anurupa Shrestha, Stephen Greszler, Steven Van Der Plas, Koen Jansen, Pieter Claes, Mia Jans, Maarten Gees, Monica Borgonovi, Gert De Wilde, Katja Conrath
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. Such correctors can have different mechanisms of action. Combinations may result in a further improved therapeutic benefit...
March 25, 2024: Journal of Medicinal Chemistry