Cystic Fibrosis Gene Therapy

With gene therapy, treatment targets the cause of cystic fibrosis rather than just treating the symptoms. Although the first gene therapy experiments have involved lung cells, scientists hope that these technologies will be adapted to treat other organs affected by cystic fibrosis.

Is There a "Cystic Fibrosis Gene?"

The cause of cystic fibrosis (CF) is a defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

This gene for cystic fibrosis makes a protein that controls the movement of salt and water in and out of your cells. In people with cystic fibrosis, the gene does not work effectively. As a result, cells that line the passageways of the lungs, pancreas, and other organs produce abnormally thick, sticky mucus. This mucus obstructs the airways and glands, which causes the characteristic signs and symptoms of cystic fibrosis.

Other factors may influence the course of cystic fibrosis. For example, changes in genes other than CFTR might help explain why some people with the disease are more severely affected than others. However, most of these genetic changes have not been identified.