Antisense oligonucleotide eluforsen improves CFTR function in F508del cystic fibrosis.

ABSTRACT:

Background

Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. In this study we assessed the effect of antisense oligonucleotide eluforsen on CFTR biological activity measured by Nasal Potential Difference (NPD) in patients with the most common mutation, F508del-CFTR.

Methods

This multi-centre, exploratory, open-label study recruited adults with CF homozygous or compound heterozygous for the F508del-CFTR mutation. Subjects received intranasal eluforsen three times weekly for 4 weeks. The primary endpoint was the within-subject change from baseline in total chloride transport (Cl-free+iso), as assessed by NPD. Secondary endpoints included within-subject change from baseline in sodium transport.

Results

In the homozygou

SUBMITTER: Sermet-Gaudelus I 

PROVIDER: S-EPMC7227803 | biostudies-literature | 2019 Jul

REPOSITORIES: biostudies-literature