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

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 with these mutations....

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Main Authors:	Marjolein M. Ensinck, Liesbeth De Keersmaecker, Anabela S. Ramalho, Senne Cuyx, Stephanie Van Biervliet, Lieven Dupont, Frauke Christ, Zeger Debyser, François Vermeulen, Marianne S. Carlon
Format:	Artigo
Language:	Inglês
Published:	European Respiratory Society 2022-04-01
Series:	ERJ Open Research
Online Access:	http://openres.ersjournals.com/content/8/2/00716-2021.full
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