Posted on: 19 December 2017, source: BioNews
A new gene therapy for haemophilia A has exceeded expectations in a recent clinical trial. All seven patients who received the high-dose therapy to correct a faulty gene for a crucial blood clotting protein showed considerable improvement. 'This is huge,' Professor John Pasi, who led the trial at Barts and Queen Mary University of London told the BBC. He added that having 'the option to think about normalising levels' of the blood clotting protein factor VIII in patients with severe haemophilia was 'mind-blowing'. Gene therapy could be a potential 'cure' for haemophilia, suggested an editorial accompanying the study published in the New England Journal of Medicine.