Improved fluorescence assays to measure the defects associated with F508del-CFTR allow identification of new active compounds

The paper Improved fluorescence assays to measure the defects associated with F508del-CFTR allow identification of new active compounds has been published by the British Journal of Pharmacology.

Cystic fibrosis (CF) is a debilitating disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which codes for a Clˉ/HCO3ˉ channel. F508del, the most common CF-associated mutation, causes both gating and biogenesis defects in the CFTR protein. This paper describes the optimisation of two fluorescence assays, capable of measuring CFTR function and cellular localisation, and their use in a pilot drug screen.

Author(s): Emily Langron, Michela I. Simone, Clémence M.S. Delalande, Jean-Louis Reymond, David L. Selwood, Paola Vergani