Article

A functional comparison of readthrough agent ELX-02 across a wide range of nonsense CFTR variants

2026-08-03

Abstract excerpt

<h4>Background</h4> Nonsense variants in CFTR account for ~10% of cystic fibrosis (CF) variants and cannot be treated with approved CFTR modulators. Translational readthrough agents such as ELX-02 offer a potential therapeutic strategy, but clinical trials evaluated mainly in G542X CFTR nonsense variant and underlined limited efficacy. This study aimed to evaluate ELX-02-mediated CFTR rescue across a broad range...

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Literature Corpus work: 304a2909-0375-553c-957f-236b68886ed4
DOI: 10.64898/2026.07.31.740556

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