If so, the gene will produce a normal protein that is vital to the health of cells lining the respiratory tract. The treatments are not expected to improve lung function yet, because only a small portion of the airways are being treated. Now Underway Several CF gene therapy clinical studies are being conducted. To date, more than 180 individuals with CF in the United States have undergone their first experimental gene therapy treatment. CF scientists at the University of Alabama at Birmingham are using liposomes as a gene delivery vehicle.
Liposomes are microscopic fat capsules.
It has proven to be safe and efficient, and has recently been modified into an aerosolized form. Initially, scientists are conducting pilot and feasibility studies to develop gene therapy technology. What's Ahead Researchers are modifying the gene delivery vectors to find other ways to circumvent the body's multi-faceted defense systems. They also must learn how long the gene treatment lasts and how often it will have to be repeated. The first gene therapy experiments involve lung cells because these cells are readily accessible and because CF lung damage is the most common life-threatening problem.