Synthesis and Biological Evaluation of Pyrazole–Pyrimidones as a New Class of Correctors of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)
Christian Vaccarin,
Guido Veit,
Tamas Hegedus
et al.
Abstract: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 the F508del-CFTR folding defect in combination with the existing
corrector drugs VX-809 and VX-661… Show more
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