A functional comparison of readthrough agent ELX-02 across a wide range of nonsense CFTR variants
Marlies Destoop, Anabela S. Ramalho, Iris A. L. Silva, Annelotte M. Vonk, Sylvia W.F. Suen, Marlou C. Bierlaagh, Sylvia F. Boj, Robert G.J. Vries, Jeffrey Matthijn Beekman, Kris De Boeck, Cornelis Korstiaan van der Ent, Margarida D. Amaral, F. Vermeulen, Sacha Spelier
This study evaluates ELX-02's effectiveness in restoring CFTR function across various nonsense variants using patient-derived intestinal organoids, finding limited and variable rescue effects.
Journal of Cystic Fibrosis · Sep 1, 2026Peer-reviewedMolecular Biology