Elexacaftor is a CFTR potentiator and acts synergistically with ivacaftor during acute and chronic treatment
Sign Up to like & getrecommendations! Published in 2021 at "Scientific Reports"
DOI: 10.1038/s41598-021-99184-1
Abstract: Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR), which lead to early death due to progressive lung disease. The development of small-molecule modulators that directly interact with CFTR… read more here.
Keywords: cftr potentiator; elexacaftor cftr; elexacaftor; acute chronic ... See more keywords
Novel CFTR modulator combinations maximise rescue of G85E and N1303K in rectal organoids
Sign Up to like & getrecommendations! Published in 2022 at "ERJ Open Research"
DOI: 10.1183/23120541.00716-2021
Abstract: Introduction Cystic fibrosis (CF) is a severe monogenic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Several types of CFTR modulators (correctors/potentiators) have been developed to overcome protein dysfunction associated… read more here.
Keywords: elexacaftor; rectal organoids; rescue; g85e n1303k ... See more keywords