In a study that could lead to new therapeutic targets for patients with cystic fibrosis (CF), a research team from the University of California-San Diego (UCSD) School of Medicine has identified a defective signaling pathway that contributes to disease severity. The researchers reported that defective signaling for a protein called peroxisome proliferator-activated receptor-γ (PPAR-γ) accounts for a portion of disease symptoms in CF, and that correction of the defective pathway with a PPAR-γ activating drug (rosiglitazone) reduces symptoms of the disease in CF mice. The scientists also showed, in both CF mice and cells from patients with CF, that the defect in signaling for PPAR-γ results from reduced levels of prostaglandins that normally activate the receptor. "The finding of the reduced PPAR-γ activating prostaglandins in CF is exciting since it could serve as a marker to identify which patients might benefit from treatment with PPAR-γ activating drugs," said Dr. Christopher Glass, senior author of the study. CF is reportedly the most common, potentially lethal genetic disease among whites, occurring in one in 3,000 births. The disease is a multisystem condition that leads to progressive lung failure, pancreatic failure, and gastrointestinal obstruction, or blockage. Exactly how the disease process occurs has been a matter of intense scientific investigation; yet despite numerous therapeutic advances, individuals with the disease continue to endure shortened lifespans. "Someone born in the 1990s with CF is expected to live to an age of around 40," said Dr Gregory Harmon, the lead author of the current study. Former NFL quarterback Boomer Esiason (photo) has a son with CF and Mr. Esiason has been very supportive of efforts to treat and cure the disease.
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