The future of cystic fibrosis care: a global perspective
Executive summary The past six decades have seen remarkable improvements in health
outcomes for people with cystic fibrosis, which was once a fatal disease of infants and young …
outcomes for people with cystic fibrosis, which was once a fatal disease of infants and young …
[HTML][HTML] CFTR modulator theratyping: Current status, gaps and future directions
JP Clancy, CU Cotton, SH Donaldson… - Journal of Cystic …, 2019 - Elsevier
Background New drugs that improve the function of the cystic fibrosis transmembrane
conductance regulator (CFTR) protein with discreet disease-causing variants have been …
conductance regulator (CFTR) protein with discreet disease-causing variants have been …
[HTML][HTML] SARS-CoV-2 reverse genetics reveals a variable infection gradient in the respiratory tract
The mode of acquisition and causes for the variable clinical spectrum of coronavirus disease
2019 (COVID-19) remain unknown. We utilized a reverse genetics system to generate a …
2019 (COVID-19) remain unknown. We utilized a reverse genetics system to generate a …
[HTML][HTML] Airway stem cell reconstitution by the transplantation of primary or pluripotent stem cell-derived basal cells
Life-long reconstitution of a tissue's resident stem cell compartment with engrafted cells has
the potential to durably replenish organ function. Here, we demonstrate the engraftment of …
the potential to durably replenish organ function. Here, we demonstrate the engraftment of …
Localization of secretory mucins MUC5AC and MUC5B in normal/healthy human airways
Rationale: MUC5AC and MUC5B are the predominant gel-forming mucins in the mucus
layer of human airways. Each mucin has distinct functions and site-specific expression …
layer of human airways. Each mucin has distinct functions and site-specific expression …
[HTML][HTML] Derivation of airway basal stem cells from human pluripotent stem cells
FJ Hawkins, S Suzuki, ML Beermann, C Barillà… - Cell Stem Cell, 2021 - cell.com
The derivation of tissue-specific stem cells from human induced pluripotent stem cells
(iPSCs) would have broad reaching implications for regenerative medicine. Here, we report …
(iPSCs) would have broad reaching implications for regenerative medicine. Here, we report …
Ion channel modulators in cystic fibrosis
M Gentzsch, MA Mall - Chest, 2018 - Elsevier
Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance
regulator (CFTR) gene and remains one of the most common life-shortening genetic …
regulator (CFTR) gene and remains one of the most common life-shortening genetic …
[HTML][HTML] IL-1β dominates the promucin secretory cytokine profile in cystic fibrosis
Cystic fibrosis (CF) lung disease is characterized by early and persistent mucus
accumulation and neutrophilic inflammation in the distal airways. Identification of the factors …
accumulation and neutrophilic inflammation in the distal airways. Identification of the factors …
Long-term differentiating primary human airway epithelial cell cultures: how far are we?
Z Bukowy-Bieryłło - Cell Communication and Signaling, 2021 - Springer
Background Human airway epithelial (HAE) cellular models are widely used in applicative
studies of the airway physiology and disease. In vitro expanded and differentiated primary …
studies of the airway physiology and disease. In vitro expanded and differentiated primary …
Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators
IM Pranke, A Hatton, J Simonin, JP Jais… - Scientific reports, 2017 - nature.com
Clinical studies with modulators of the Cystic Fibrosis Transmembrane conductance
Regulator (CFTR) protein have demonstrated that functional restoration of the mutated …
Regulator (CFTR) protein have demonstrated that functional restoration of the mutated …