Gene Therapy For Cystic Fibrosis Shows Beneficial Effect On Lung Function

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. “Patients who received the gene therapy showed a significant, if modest, benefit in tests of lung function compared with the placebo group and there were no safety concerns,” said senior author Professor Eric Alton from the National Heart and Lung Institute at Imperial College London. “Whilst the effect was inconsistent, with some patients responding better than others, the results are encouraging.” read more

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