Article
The preclinical discovery and development of the combination of ivacaftor + tezacaftor used to treat cystic fibrosis.
Expert opinion on drug discovery - 1 Aug 2020
Guerra Lorenzo, Favia Maria, Di Gioia Sante, Laselva Onofrio, Bisogno Arianna, Casavola Valeria, Colombo Carla, Conese Massimo
Abstract excerpt
INTRODUCTION: Cystic Fibrosis (CF) is caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. The most common mutation, F508del, induces protein misprocessing and loss of CFTR function. The discovery through in vitro studies of the CFTR correctors (i.e. lumacaftor, tezacaftor) that partially rescue the misprocessing of F508del-CFTR with the potentiator ivacaftor is promising in...
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