Adenine base editing of CFTR using receptor targeted nanoparticles restores function to G542X cystic fibrosis airway epithelial cells

Abstract The cystic fibrosis (CF) causing variant G542X harbours a premature translation stop signal in the cystic fibrosis transmembrane conductance regulator (CFTR) mRNA. This results in nonsense-mediated decay and loss of functional CFTR protein which leads to defective anion transport and the de...

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Bibliographic Details

Published in:	Cellular and Molecular Life Sciences
Main Authors:	Isabelle Rose, Miriam Greenwood, Matthew Biggart, Natalie Baumlin, Robert Tarran, Stephen L. Hart, Deborah L. Baines
Format:	Article
Language:	English
Published:	Springer 2025-04-01
Subjects:	Class 1 mutation Transfection Air–liquid-interface Nasal Ciliated cell Secretory cell
Online Access:	https://doi.org/10.1007/s00018-025-05587-y