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Gene therapy trial for cystic fibrosis shows modest benefits

BMJ 2015; 351 doi: (Published 03 July 2015) Cite this as: BMJ 2015;351:h3608
  1. Nigel Hawkes
  1. 1London

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.

The improvement was just 3.7%—insufficient to justify the treatment’s use in clinical practice but hugely encouraging to the UK Cystic Fibrosis Gene Therapy Consortium, which ran the trial in 18 …

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