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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Detalles Bibliográficos
Autores principales: Ensinck, Marjolein M., De Keersmaecker, Liesbeth, Ramalho, Anabela S., Cuyx, Senne, Van Biervliet, Stephanie, Dupont, Lieven, Christ, Frauke, Debyser, Zeger, Vermeulen, François, Carlon, Marianne S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: European Respiratory Society 2022
Materias:
Original Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9016267/
https://www.ncbi.nlm.nih.gov/pubmed/35449760
http://dx.doi.org/10.1183/23120541.00716-2021

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