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Gene therapy: a new Rx for CF?

Cystic fibrosis patients are inhaling an experimental treatment and hoping for the best: genes that cure their disease.

Some 30,000 Americans may soon breathe easier. Doctors are closing in on a cure for their disease, cystic fibrosis (CF), the most common fatal genetic disorder in the country.

Using a new technique called gene therapy, physicians at the National Heart, Lung, and Blood Institute in Bethesda, MD, hope to go beyond just treating the symptoms of CF. They aim to fix CF's root cause: a faulty gene.

The faulty gene causes a thick mucus buildup in the lungs and other organs. Breathing is often a chore for many CF patients, who must endure daily "clappings" on the back to dislodge the mucus and prevent organ-damaging infections. Without a new treatment--such as healthy genes delivered into their cells through gene therapy--most CF patients will not live past age 30.


Since April, Dr. Ronald Crystal, who heads the research, has treated three CF patients using the new therapy. His gene-insertion device: a common cold virus loaded with a healthy copy of the CF gene.

Like a regular cold virus, the virus-gene combo travels into airway cells (see diagram). But instead of giving the patients colds, the altered virus gives them the genetic ammo they need to fight their disease. If all goes well, the virus will insert a healthy copy of the gene. The new gene should restore normal cell function and help clear up the mucus, explains Crystal.

Such a cure seemed far off until 1989, when scientists discovered the faulty CF gene (see SW 1/26/90, p. 7) and learned how it works: It prevents chloride molecules from flowing-out of cells. As the chloride level inside a cell rises, water from surrounding lung tissue surges in to dilute it, leaving behind thick, dehydrated mucus. The mucus clogs various organs, trapping infection-causing bacteria.


Last year, researchers successfully used gene therapy to eliminate symptoms in CF-infected rats. Now, for the first time, the virus-gene combo is being tested in humans. "Our first goal is to demonstrate that the technique is three subjects are doing well, he says. Seven others are expected to receive treatment soon.

If the gene therapy works, Crystal remarks, "we're on the way to a cure." Says Robert Beall of the Cystic Fibrosis Foundation, "For the first time, we're not just treating the symptoms of the disease. We're getting at its root cause." And for CF patients, that's cause for relief.
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Title Annotation:experimental cystic fibrosis treatment
Author:Freiman, Chana
Publication:Science World
Date:Oct 8, 1993
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