Novel CFTR modulator combinations maximise rescue of G85E and N1303K in rectal organoids
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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