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Encouraging results from Cystic Fibrosis gene therapy trial

An early-stage trial of a new gene therapy for people with Cystic Fibrosis has delivered what scientists claim are encouraging results.

RTE News, Jul 3, 2015

The gene-altering medicine was shown to moderately improve or stabilise the lung function of patients and lead to health gains.

Cystic Fibrosis is an inherited chronic disease affecting the lungs and digestive system of 1,200 children and adults in Ireland.

It is caused by a defective gene, which leads the body to produce thick mucus that clogs the lungs, leading to life-threatening infections.

The trial of the new gene therapy was conducted by scientists in London and Edinburgh, and involved 136 CF patients aged 12 and over.

The participants inhaled fat droplets carrying genetic material into their lungs every month for a year.

The globules fused with fatty cell membrane walls, allowing DNA slip into the lungs.

The results, published in the journal Lancet Respiratory Medicine, show lung function was almost 4% better in those taking the therapy, while participants with the worst lung function at the start of the study experienced a 6.4% gain.

The scientists say while modest, the improvements were significant and a world first.

Speaking on RTEs Morning Ireland, Professor Deborah Gill, co-author of trial, said the treatment could be life-changing for patients.

She said that this was the first time that lung function improved in CF sufferers.

Ms Gill said: "The particular good thing about gene therapy is it doesn't matter what kind of mutation that you have. Many of the new drugs on the market for Cystic Fibrosis, and it is an exciting time for treatment, are actually only useful for patients with certain mutations."

"Gene therapy can theoretically help all patients with all mutations," she said.

Ms Gill explained that the treatment works by replacing the mutated gene in the lung with a copy of the gene that functions normally.

She also said that the cost of the treatment is not yet known.

Ms Gill also cautioned that a lot more work is required before the therapy could become a viable clinical treatment.

That work begins with a much larger study starting next year.

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