Author

Margarida D. Amaral

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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

Review Open access Aug 2026

Cystic Fibrosis and Colorectal Cancer Risk: Reprogramming of the Intestinal Epithelial Niche and Cell‐State Plasticity in the CFTR Modulator Era

It is proposed that CF may provide a naturally occurring human context in which chronic epithelial stress sustains EMP‐like pressure, a concept that requires direct validation in human CF intestinal tissue.

B. Umashankar, Ines Pankonien, Margarida D. Amaral et al. · Cell Proliferation · 0 citations

#gene editing Review Open access Aug 2026

Combining Gene Therapy with Current Modulator Treatments for Cystic Fibrosis: A Promising Area of Research

A potential combination between gene therapy approaches and existing modulators to improve treatment eligibility, safety, and efficacy for cystic fibrosis patients is discussed.

Xavier Buin, Rosy Ghanem, Ines Pankonien et al. · Pharmaceutics · 0 citations