Article
Functional Restoration of CFTR Nonsense Mutations in Intestinal Organoids.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Mar 2022
de Poel E, Spelier S, Suen S W F, Kruisselbrink E, Graeber S Y, Mall M A, Weersink E J M, van der Eerden M M, Koppelman G H, van der Ent C K, Beekman J M
Abstract excerpt
BACKGROUND: Pharmacotherapies for people with cystic fibrosis (pwCF) who have premature termination codons (PTCs) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene are under development. Thus far, clinical studies focused on compounds that induce translational readthrough (RT) at the mRNA PTC location. Recent studies using primary airway cells showed that PTC functional restoration can be...
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