---
title: Application of HUB Organoids in cystic fibrosis
description: A new peer-reviewed publication demonstrates the application of HUB Organoids to advancing cystic fibrosis treatment
image: https://resources.huborganoids.nl/hubfs/publication-thumb-targeting-g542x-cftr.png
---

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

J Cyst Fibros | May 2021

[view publication](https://pubmed.ncbi.nlm.nih.gov/33558100/)

![](https://resources.huborganoids.nl/hubfs/publication-thumb-targeting-g542x-cftr.png)

## Abstract

Promoting full-length protein production is a requisite step to address some of the remaining unmet medical needs 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 mediated read-through using the CFTR-dependent Forskolin-induced swelling (FIS) assay across a selection of G542X genotype patient-derived organoids (PDOs).

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