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 …

Microfluidic lumen-based systems for advancing tubular organ modeling

M Virumbrales-Muñoz, JM Ayuso, MM Gong… - Chemical Society …, 2020 - pubs.rsc.org
Microfluidic lumen-based systems are microscale models that recapitulate the anatomy and
physiology of tubular organs. These technologies can mimic human pathophysiology and …

[HTML][HTML] Comparison of various cell lines and three-dimensional mucociliary tissue model systems to estimate drug permeability using an in vitro transport study to …

T Furubayashi, D Inoue, N Nishiyama, A Tanaka… - Pharmaceutics, 2020 - mdpi.com
Recently, various types of cultured cells have been used to research the mechanisms of
transport and metabolism of drugs. Although many studies using cultured cell systems have …

Antisense oligonucleotide-mediated correction of CFTR splicing improves chloride secretion in cystic fibrosis patient-derived bronchial epithelial cells

WE Michaels, RJ Bridges… - Nucleic Acids Research, 2020 - academic.oup.com
Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CF
transmembrane conductance regulator (CFTR) gene, encoding an anion channel that …

[HTML][HTML] Cystic fibrosis transmembrane conductance regulator dysfunction in platelets drives lung hyperinflammation

G Ortiz-Muñoz, AY Michelle… - The Journal of …, 2020 - Am Soc Clin Investig
Cystic fibrosis (CF) lung disease is characterized by an inflammatory response that can lead
to terminal respiratory failure. The cystic fibrosis transmembrane conductance regulator …

Antibiotic treatment for Burkholderia cepacia complex in people with cystic fibrosis experiencing a pulmonary exacerbation

R Lord, AM Jones, A Horsley - Cochrane Database of …, 2020 - cochranelibrary.com
Background Chronic pulmonary infection is a hallmark of lung disease in cystic fibrosis.
Infections dominated by organisms of the Burkholderia cepacia complex, a group of at least …

Recombinant acid ceramidase reduces inflammation and infection in cystic fibrosis

AI Gardner, IJ Haq, AJ Simpson, KA Becker… - American Journal of …, 2020 - atsjournals.org
Rationale: In cystic fibrosis the major cause of morbidity and mortality is lung disease
characterized by inflammation and infection. The influence of sphingolipid metabolism is …

[HTML][HTML] Impact of bacterial toxins in the lungs

R Lucas, Y Hadizamani, J Gonzales, B Gorshkov… - Toxins, 2020 - mdpi.com
Bacterial toxins play a key role in the pathogenesis of lung disease. Based on their structural
and functional properties, they employ various strategies to modulate lung barrier function …

[HTML][HTML] Small molecule anion carriers correct abnormal airway surface liquid properties in cystic fibrosis airway epithelia

A Gianotti, V Capurro, L Delpiano… - International Journal of …, 2020 - mdpi.com
Cystic fibrosis (CF) is a genetic disease characterized by the lack of cystic fibrosis
transmembrane conductance regulator (CFTR) protein expressed in epithelial cells. The …

Efficient RNP-directed human gene targeting reveals SPDEF is required for IL-13–induced mucostasis

KD Koh, S Siddiqui, D Cheng, LR Bonser… - American Journal of …, 2020 - atsjournals.org
Primary human bronchial epithelial cell (HBEC) cultures are a useful model for studies of
lung health and major airway diseases. However, mechanistic studies have been limited by …