A functional comparison of readthrough agent ELX-02 across a wide range of nonsense CFTR variants. (PubMed, J Cyst Fibros) - Sep 12, 2026 - "ELX-02 induces limited and highly heterogeneous CFTR rescue across nonsense variants. PDIO-based functional screening provides a framework to guide patient selection and stratification for future readthrough therapy trials."
Journal • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases
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; Anabela S Ramalho; Annelotte M Vonk; Cornelis K van der Ent; François Vermeulen; Iris A L Silva; Jeffrey M Beekman; Kris De Boeck; Margarida D Amaral; Marlies Destoop; Marlou C Bierlaagh; Robert G J Vries; Sacha Spelier; Sylvia F Boj; Sylvia Suen
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