Cystic Fibrosis

There's still no cure for cystic fibrosis, but scientists think they may be homing in on new treatments for the deadly hereditary disease - thanks to new research that pinpoints the mutant genes responsible for severe CF.
Researchers studied DNA from almost 3,500 cystic fibrosis patients, searching for mutant genes shared by those with the most severe forms of severe cystic fibrosis. They found what they were looking for on chromosomes 11 and 20 - two of the 46 chromosomes that make up the human genome.
"We already know which gene causes CF, but to a large extent that gene does not by itself explain how severe the condition will be," study author Dr. Garry Cutting, professor of pediatrics at Johns Hopkins Institute for Genetic Medicine, said in a written statement. "Now we've discovered new genes that influence the course of disease and may enable prediction of disease severity and, most importantly, customization of treatments for patients" who have the worrisome genes.

Worrisome, indeed. Cystic fibrosis causes scarring through the body, especially the lungs and the pancreas. Symptoms include belly pain, coughing, bloating, constipation, and nausea. Cutting said most cystic fibrosis patients born today are unlikely to live past their mid-thirties - with some succumbing before they reach their teens and others living into their fifties.
The goal, Cutting said, is to find treatments that will enable people with severe cystic fibrosis live as long as those with milder forms of the disease.  "And this is the first step."