Cistična fibroza (CF) je najčešća autosomna recesivna bolest u zapadnim zemljama. Kronične upale i povećana podložnost infekcijama s patogenima poput Pseudomonas aeruginosa su glavne karakteristike cistične fibroze. Pokazano je da ceramid ima centralnu ulogu u različitim plućnim infekcijama, uključujući infekcije s Pseudomonas aeruginosa, te da se nakuplja u plućnom tkivu i stanicama oboljelih od cistične fibroze. Nadalje, ekspresija sfingozina je značajno reducirana u dišnim putevima miševa i ljudi oboljelih od cistične fibroze. No, o ulozi glukozilceramida u cističnoj fibrozi jako se malo zna. U ovom radu pokazujem da su razine glukozilceramida reducirane na apikalnoj površini bronhijalnih stanica i stanica dušnika u CF mišu. Inhalacija CF miševa s glukozilceramidom je zaštitila miševe od infekcije s Pseudomonas aeruginosa dok su ne-inhalirani CF miševi razvili tešku upalu pluća. In vitro studije nisu pokazale direktan učinak glukozilceramida na Pseudomonas aeruginosa. Ovi rezultati indiciraju da glukozilceramid ima potencijal postati nova meta u liječenju cistične fibroze.

Cystic fibrosis (CF) is the most common autosomal recessive disorder in Western countries and it is characterized by chronic inflammation and increased susceptibility to infection by pathogens such as Pseudomonas aeruginosa. It has been previously shown that ceramide has a central role in various pulmonary infections, including infections with P. aeruginosa, and is accumulated in lung tissues and cells from CF patients. In addition, sphingosine is severely down-regulated in the airways of CF mice and patients. However, little is known about a potential role of glucosylceramide in cystic fibrosis. Here I demonstrate that levels of glucosylceramide are reduced on the apical surface of bronchial and tracheal epithelial cells in CF mice. Inhalation of CF mice with glucosylceramide protected these mice from infection with P. aeruginosa, while non-inhaled CF mice developed severe pneumonia. However, in vitro studies failed to show a direct effect of glucosylceramide on Pseudomonas aeruginosa. This data indicate that glucosylceramide has a potential as a novel target to treat CF.