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.

This study develops a method to differentiate human nasal epithelial cells into airway-like tissues using small molecules, creating disease models for respiratory conditions.

The paper demonstrates that ACE-tRNAs can suppress nonsense mutations in CF by rescuing CFTR transcript abundance and channel function in multiple models.

The paper screens 1400 FDA-approved drugs in CF patient-derived intestinal organoids using a functional assay to identify potential repurposing for rare CF genotypes.

This paper introduces a fluorescent reporter system for evaluating and enhancing prime editing efficiency in genomic targets.