Effect of CFTR correctors on the traffic and the function of intracellularly retained ABCB4 variants.

Amel Ben Saad Virginie Vauthier Ágota Tóth Angelika JanaszkiewiczAnne-Marie Durand-SchneiderAlix Bruneau Jean-Louis Delaunay Martine Lapalus Elodie MareuxIsabelle GarcinEmmanuel M Gonzales Chantal Housset Tounsia Aït-Slimane Emmanuel Jacquemin Florent Di Meo Thomas Falguières

Published in: Liver international : official journal of the International Association for the Study of the Liver (2021)

Our results illustrate the uncoupling between the traffic and the activity of ABCB4 because the same molecules can rescue the traffic of defective variants while they inhibit the secretion activity of the transporter. We expect that this study will help to design new pharmacological tools with potential clinical interest.

Keyphrases

air pollution
copy number
cystic fibrosis
gene expression
nitric oxide