Scientists from the University of California, San Diego School of Medicine, have identified potential targets for treatment of patients with cystic fibrosis.
They have discovered a defective signaling pathway that contributes to disease severity.
In the study, researchers found that defective signaling for a protein called the peroxisome proliferator-activated receptor-? (PPAR-?) accounts for a portion of disease symptoms in cystic fibrosis, and that correction of the defective pathway reduces symptoms of the disease in mice.
Lead author Gregory Harmon, study supervisor Christopher Glass, and colleagues showed that both mice and cells from patients with cystic fibrosis have a defect in signaling for PPAR-?, as a result of reduced levels of prostaglandins that activate the receptor.
Cystic fibrosis is a multisystem condition that leads to progressive lung failure, pancreatic failure and gastrointestinal obstruction, or blockage.
The study has been published in the journal Nature Medicine.