12Feb 2021
Targeting G542X CFTR nonsense alleles with ELX-02 restores CFTR function in human-derived intestinal organoids

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

Advances in cystic fibrosis (CF) patient respiratory care, nutrition, and new treatment options in recent years have improved quality of life for many patients. However, the disease-modifying therapies available target the protein itself and are therefore not applicable to those individuals with alleles incapable of producing protein at all, including those with nonsense alleles.

  • #pulmonology

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