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

Pub. Date : 2021 May

PMID : 33558100






2 Functional Relationships(s)
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1 Here we evaluate ELX-02 mediated read-through using the CFTR-dependent Forskolin-induced swelling (FIS) assay across a selection of G542X genotype patient derived organoids (PDOs). Colforsin CF transmembrane conductance regulator Homo sapiens
2 RESULTS: PDOs homozygous for G542X or heterozygous with a second minimally functional allele had significantly increased CFTR activity with ELX-02 in a dose-dependent fashion across a variety of forskolin induction concentrations. Colforsin CF transmembrane conductance regulator Homo sapiens