Article
Synthesis and Biological Evaluation of Pyrazole-Pyrimidones as a New Class of Correctors of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR).
Journal of medicinal chemistry - 22 Aug 2024
Vaccarin Christian, Veit Guido, Hegedus Tamas, Torres Odalys, Chilin Adriana, Lukacs Gergely L, Marzaro Giovanni
Abstract excerpt
Cystic fibrosis (CF) is caused by the functional expression defect of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Despite the recent success in CFTR modulator development, the available correctors only partially restore the F508del-CFTR channel function, and several rare CF mutations show resistance to available drugs. We previously identified compound 4172 that synergistically rescued...
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