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Organoid platform for the development of innovative treatments for Cystic Fibrosis

Cystic fibrosis (CF) is the most common monogenic fatal disorder in the European population, with a prevalence of approximately 1 out of 2500 live births. CF is caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene, which encodes a chloride channel expressed in the apical membrane of epithelial cells. To date, almost 2000 CFTR mutations have been described in the CF database, Phe508del being the most common mutation worldwide. Despite extensive efforts treatment for CF patients remain symptomatic without targeting the underlying cause of the disease resulting in a high disease burden for the patients. Within this project the consortium will establish a CF technology platform focusing on rare CF mutations and built on human intestinal organoids and specific mouse models. This platform will be the basis for developing innovative personalized therapies for CF.
Date:1 Oct 2015  →  30 Sep 2017
Keywords:Cystic Fibrosis
Disciplines:Respiratory medicine