Cystic fibrosis remains the most common fatal genetic disease in Europe. Advanced medical treatment has raised the average life span to 30 years. Treatments so far manage acute symptoms and slow the disease progression. Gene therapy may open the door for further progress.
Disease: Cystic Fibrosis
Last update: June 2008
Intro
FAQ: What is it?
Cystic Fibrosis results from a defect in a single gene that makes a protein called CFTR ('cystic fibrosis transmembrane conductance regulator'). This defect results in the secretion of thick, sticky mucus in the lungs and glands of the body. It is the most common fatal hereditary disease in Caucasians.