Gene therapy trial for cystic fibrosis shows modest benefits

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A gene therapy trial has shown a small but notable health benefit in people with cystic fibrosis—proof of principle that, after a long wait, hopes of effective treatments for inherited diseases may one day be realised.

In a trial involving 136 cystic fibrosis patients aged over 12 who used nebulisers to inhale the correct gene wrapped in an envelope of fat, modest benefits were observed over a range of measurements when compared with patients inhaling a placebo. The main aim was to show that the treatment, given monthly for a year, would improve lung function measured by the volume of air the patients could expel in one second.