Article

The behaviour of the cystic fibrosis respiratory epithelium and its response to multidose CFTR gene therapy

2016-01-01

Abstract excerpt

Cystic fibrosis (CF) is a clinical syndrome resulting from inherited mutations to the cystic fibrosis transmembrane conductance regulator (CFTR) protein, whose absent or reduced function results in abnormal epithelial ion transport and an abnormal transepithelial potential difference (PD), leading to downstream epithelial dysfunction and a pathognomonic clinical phenotype. Most treatments to date manage the diseas...

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Literature Corpus work: bdec4831-cc3a-5441-bdd4-76e34ac3bb1f
DOI: 10.25560/41881

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