Proceedings of the National Academy of Sciences of the United States of AmericaCystic Fibrosis Transmembrane Conductance RegulatorHumansQuinolonesCystic FibrosisAminophenols
Chronic and acute modulator treatment restore wild-type-like activity and stability to the primary cystic fibrosis-causing CFTR variant.
Mayuree Rodrat, James N Charlick, David N Sheppard
Published: 202610.1073/pnas.2523950123
Abstract
Elexacaftor-tezacaftor-ivacaftor has transformed the treatment of people with cystic fibrosis (CF) and the predominant cystic fibrosis transmembrane conductance regulator (CFTR) variant F508del by delivering the faulty channel to the plasma membrane…
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