Current biochemical approaches in cystic fibrosis research.

The clinical abnormalities in cystic fibrosis can be categorized into three headings: 
pulmonary disease, digestive disorders, and ion transport abnormalities. Research directions in 
cystic fibrosis have evolved from observations of the characteristic malfunctions inherent 
in the disease. These include defects in mucociliary function, mucous secretion, 
membrane transport and glycoprotein biosynthesis. Drawing from what is known about 
the bases of other inherited diseases, one could predict that cystic 
fibrosis results from an abnormal functional protein, as in sickle cell 
anemia; an enzyme deficiency, as in phenylketonuria; or a deficiency of 
a normal inhibitor, as in hereditary angioedema. 