A new Inserm study published in Nature Communications opens a path to a therapy that would allow patients with cystic fibrosis to recover their respiratory function. Researchers have indeed succeeded in highlighting a new mechanism making it possible to restore the functioning of a cell channel located in particular in the pulmonary mucosa.
An assay on mice and cell cultures
Cystic fibrosis is a serious genetic disease affecting digestive and pulmonary functions. It is linked to the deficiency of an extremely unstable gene, located on the
chromosome 7. And because of the mutation of this gene, the CFTR protein, which functions as a channel for the exchange of chloride ions between the interior and exterior of cells, is deficient. To date, more than 2000 gene mutations have been identified, which complicates therapeutic approaches.
In Inserm research, this exchange function has only been restored in mice and in cell cultures from patients with cystic fibrosis. Corn “such a strategy would ultimately target all patients regardless of the mutation and correct major parameters in the development of the pathophysiology of patients with cystic fibrosis “ said Olivier Tabary, lead author of the study.
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