Origins of cystic fibrosis lung disease

DA Stoltz, DK Meyerholz, MJ Welsh - New England Journal of …, 2015 - Mass Medical Soc
Mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator
undermine many host defense systems by inhibiting the function of airway-surface liquid …

The innate immune function of airway epithelial cells in inflammatory lung disease

PS Hiemstra, PB McCray, R Bals - European respiratory …, 2015 - Eur Respiratory Soc
The airway epithelium is now considered to be central to the orchestration of pulmonary
inflammatory and immune responses, and is also key to tissue remodelling. It acts as the first …

[HTML][HTML] Transgenic ferret models define pulmonary ionocyte diversity and function

F Yuan, GN Gasser, E Lemire, DT Montoro… - Nature, 2023 - nature.com
Speciation leads to adaptive changes in organ cellular physiology and creates challenges
for studying rare cell-type functions that diverge between humans and mice. Rare cystic …

Cystic fibrosis: emergence of highly effective targeted therapeutics and potential clinical implications

MA Mall, N Mayer-Hamblett… - American journal of …, 2020 - atsjournals.org
Cystic fibrosis (CF) remains the most common life-shortening hereditary disease in white
populations, with high morbidity and mortality related to chronic airway mucus obstruction …

CFTR: cystic fibrosis and beyond

MA Mall, D Hartl - 2014 - Eur Respiratory Soc
Cystic fibrosis (CF) remains the most common fatal hereditary lung disease. The discovery of
the cystic fibrosis transmembrane conductance regulator (CFTR) gene 25 years ago set the …

Inflammation and its genesis in cystic fibrosis

DP Nichols, JF Chmiel - Pediatric pulmonology, 2015 - Wiley Online Library
The host inflammatory response in cystic fibrosis (CF) lung disease has long been
recognized as a central pathological feature and an important therapeutic target. Indeed …

In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model of cystic fibrosis

X Sun, Y Yi, Z Yan, BH Rosen, B Liang… - Science translational …, 2019 - science.org
Cystic fibrosis (CF) is a multiorgan disease caused by mutations in the cystic fibrosis
transmembrane conductance regulator (CFTR). In patients with CF, abnormalities initiate in …

Gene therapy for cystic fibrosis: new tools for precision medicine

JA Lee, A Cho, EN Huang, Y Xu, H Quach, J Hu… - Journal of Translational …, 2021 - Springer
The discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for incredible
progress in treating the disease such that the mean survival age of individuals living with CF …

Cystic fibrosis gene therapy: looking back, looking forward

AL Cooney, PB McCray Jr, PL Sinn - Genes, 2018 - mdpi.com
Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic
fibrosis transmembrane conductance regulator (CFTR) gene that encodes a cAMP …

The microbiome in cystic fibrosis pulmonary disease

A Françoise, G Héry-Arnaud - Genes, 2020 - mdpi.com
Cystic fibrosis (CF) is a genetic disease with mutational changes leading to profound
dysbiosis, both pulmonary and intestinal, from a very young age. This dysbiosis plays an …