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Gene Therapy For Cystic Fibrosis

Cystic fibrosis is an inherited disease in which the body's mucus becomes so thick and sticky that it prevents normal functioning. Treatment with new antibiotics now allows many children to reach adulthood, but there is no cure.// New research is moving us slightly closer to a treatment for cystic fibrosis that modifies the abnormal gene that causes the disease.

Working with mice, scientists have restored key chemicals in cells to levels that should ease symptoms of the fatal disease. Still, it's too early to know whether the same technique will work in people. In this study, scientists at the University of Iowa used a harmless virus to carry a corrected genetic information into the gene associated with cystic fibrosis. These "corrected" cells then were able to process a normal version of a protein that helps regulate mucus in the body.

Although the results were encouraging, researcher John F. Engelhardt notes the experiment must be modified before it can be tried in people. Another virus "carrier" must be found because the one used in the mouse experiments doesn't easily enter human cells. Also, it's important to remember that the mouse experiment did not cure cystic fibrosis, but it did show promise of easing symptoms.

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