Twenty six years ago, the gene responsible for cystic fibrosis was identified. Now, researchers have shown that people with the lung-damaging condition can benefit from gene therapy.
The trial involved 116 participants, 62 of whom received at least 9 monthly doses of the therapy, while a control group of 54 inhaled saline solution. To gauge the effect on lung functioning, changes in the volume of air each participant could force out of their lungs in one second were tracked.
At the end of 12 months, those who had been breathing in the gene treatment had, on average, 3.7 per cent better lung function than those on the placebo.
“While this is obviously not a home run, I do think the results are impressive. I’m pleased that gene therapy has been shown to safely deliver statistically significant treatment effects,” says Oli Rayner, who has cystic fibrosis and works as an advocate for those who have the disease in the UK.
Source: New Scientist