LONDON — Scientists conducting a major trial of a therapy that replaces the faulty gene responsible for cystic fibrosis say the treatment has showed significant benefit for the first time in patients' lung function. The technique, developed with the technology commercialization firm Imperial Innovations, replaces the defective gene behind the inherited lung disease 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,” said Eric Alton, a professor at Imperial College London who led the trial. Briefing reporters about the results, he said they were “encouraging” but cautioned that because the effect was inconsistent, with some patients responding better than others, the gene therapy was not ready for regular clinical use. “The effects were modest and variable,” he stressed. — Reuters
