Targeting G542X CFTR nonsense alleles with ELX-02 restores CFTR function in human-derived intestinal organoids.

Abstract excerpt

BACKGROUND: Promoting full-length protein production is a requisite step to address some of the remaining unmet medical need for those with Cystic Fibrosis (CF) nonsense alleles. ELX-02 promotes read-through of mRNA transcripts bearing nonsense mutations, including the most common CF nonsense allele G542X, in several different preclinical models including human bronchial epithelial cells. Here we evaluate ELX-02...

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• Alleles
• Cells, Cultured
• Codon, Nonsense
• Cystic Fibrosis
• Cystic Fibrosis Transmembrane Conductance Regulator