The U UGA C sequence provides a favorable context to ELX-02 induced CFTR readthrough.

Authors :: Pranke IM
Varilh J
Hatton A
Faucon C
Girodon E
Dreano E
Chevalier B
Karri S
Reix P
Durieu I
Bidou L
Namy O
Taulan M
Hinzpeter A
Sermet-Gaudelus I
Source :: Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society [J Cyst Fibros] 2023 May; Vol. 22 (3), pp. 560-563. Date of Electronic Publication: 2022 Nov 16.
Publication Year :: 2023
Abstract: Competing Interests: Conflict of Interest Iwona M. Pranke, Jessica Varilh, Aurélie Hatton, Caroline Faucon Emmanuelle Girodon, Elise Dreano, Benoit Chevalier, Sabrina Karry(,) Philippe Reix, Isabelle Durieu, Laure Bidou, Olivier Namy, Magali Taulan, Alexandre Hinzpeter have no conflict of interest. Isabelle Sermet-Gaudelus has received Vertex Innovation Award from Vertex therapeutics and participated to Scientific Advisory Boards (Vertex therapeutics, Eloxx Therapeutics).

Subjects :: Humans
Codon, Terminator
Furans
Cystic Fibrosis Transmembrane Conductance Regulator genetics
Cystic Fibrosis drug therapy
Cystic Fibrosis genetics

Language :: English
ISSN :: 1873-5010
Volume :: 22
Issue :: 3
Database :: MEDLINE
Journal :: Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
Publication Type :: Academic Journal
Accession number :: 36400713
Full Text :: https://doi.org/10.1016/j.jcf.2022.10.010

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