1. The U UGA C sequence provides a favorable context to ELX-02 induced CFTR readthrough.
- Author
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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, and Sermet-Gaudelus I
- Subjects
- Humans, Codon, Terminator, Furans, Cystic Fibrosis Transmembrane Conductance Regulator genetics, Cystic Fibrosis drug therapy, Cystic Fibrosis genetics
- 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).
- Published
- 2023
- Full Text
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