First trial of gene therapy for cystic fibrosis to show beneficial effect on lung function

Friday, July 3, 2015 - 08:30 in Health & Medicine

For the first time gene therapy for cystic fibrosis has shown a significant benefit in lung function compared with placebo, in a phase 2 randomized trial. The technique replaces the defective gene response for cystic fibrosis by using inhaled molecules of DNA to deliver a normal working copy of the gene to lung cells.

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