Author

A. Ramalho

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Open access Aug 2026

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

PDIO-based functional screening provides a framework to guide patient selection and stratification for future readthrough therapy trials and induces limited and highly heterogeneous CFTR rescue across nonsense variants.

M. Destoop, A. Ramalho, I. A. L. Silva et al. · bioRxiv · 0 citations