Molecular BiologyJournal of Cystic FibrosisPeer-reviewed
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…·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.
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