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Gene Therapy Can Help Treat Cystic Fibrosis Patients

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Cystic fibrosis could soon be treated with gene therapy, following a new finding. The disorder is caused by a faulty gene, and researchers are now seeing positive results in a new treatment that replaces that snippet of genetic code.

UK Cystic Fibrosis Gene Therapy Consortium, a collaboration of researchers from the Universities of Oxford and Edinburgh and Imperial College London, conducted the study.

Cystic fibrosis (CF) is a genetic disorder, also known as mucoviscidosis, which affects the lungs, along with the kidneys, liver, intestines and the pancreas. The disorder is marked by the presence of a sticky, thick mucus in lungs. Around 70,000 people worldwide have been diagnosed with the disease, 30,000 (43 percent) of which are in the United States. This mucus can both obstruct air coming into and out of the lungs, but can also result in potentially-deadly infections of the organ.

Investigators studied 116 patients over the age of 12. While one group of patients received the gene-replacement therapy, a control group was provided with a placebo. Subjects in the experimental group were provided with around one teaspoon of the treatment each month. Investigators found that the new treatment did bring about some improvement in lung function.

"This clinical trial is the first time that gene therapy has been shown to have a significant benefit in lung function. We have spent several years working to improve the gene therapy — specifically the plasmid molecule carrying the gene — to make sure the beneficial effects could last a long time, rather than only the few days that had been observed in earlier trials," Deborah Gill from the Radcliffe Department of Medicine at Oxford University said.

Although the results of the study were encouraging, treatment for patients with cystic fibrosis remains on the distant horizon. A significant portion of subjects provided with the drug saw no benefit from the treatment, and researchers wish to examine why this population was resistant to treatment. Future investigations will also study the effects of larger doses of the drug.

Most patients with CF are diagnosed by their second birthday. During the 1950s, very few children with the disorder lived long enough to attend school. Today, many people with the genetic disorder live to their 30s and 40s, occasionally beyond. Diagnosis is usually carried out through a sweat test.

"A mild chemical and a small amount of electricity are placed on the skin (usually on the arm) to stimulate the sweat glands. Sweat is then collected and the amount of chloride, a component of salt in the sweat, is measured. A high level of chloride means that the person has cystic fibrosis. The sweat test is painless," the Cystic Fibrosis Foundation reports.

Currently, there is no cure for cystic fibrosis, although Kalydeco was approved by the FDA in 2012 to help treat the disorder.

Photo: Stephen Weppler | Flickr

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