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Gene therapy for CF patients produces encouraging results: study

Xinhua, July 4, 2015 Adjust font size:

By replacing the faulty gene responsible for cystic fibrosis (CF), gene therapy improved the lung function of CF patients in a major UK trial, according to a study published Friday by the University of Edinburgh.

CF can cause patients' lungs to be filled with thick mucus and the patients are vulnerable to recurrent chest infections, which eventually destroy the lungs. Cystic fibrosis affects around 10,000 people in Britain and over 90,000 worldwide.

In the trial of the new treatment, 136 patients aged 12 and over received monthly doses of either the therapy or a dummy treatment for one year. Researchers from University of Edinburgh and other UK institutes participated in the study.

The results showed that patients who received the therapy had a "significant, but modest, improvement" in lung function compared with those receiving a placebo.

The gene mutation that causes cystic fibrosis was identified in 1989. This discovery opened the door to new treatments to repair the damaged gene. However, current therapies have improved life expectancy for patients but are focused on treating the symptoms of the disease rather than the cause, researchers said.

Despite the encouraging results, more research is needed to improve the effectiveness of the therapy before it will be suitable for clinical use, researchers also said.

The findings have been published in the journal The Lancet Respiratory Medicine. Endit