Jake Omer, pictured with his family, said that the gene therapy trial had changed his lifeA cure for haemophilia has moved a step closer after “mind-blowing” results from an early gene therapy trial. About 2,000 people in the UK have the most serious form, haemophilia A, which requires regular injections of a clotting protein. A defective gene means that patients do not produce the clotting protein factor VIII. Scientists have long hoped that the disease could be cured by effectively replacing the defective gene. In a British trial of the technique on 13 patients, levels of the protein returned to normal in all but two…

Source: The Times December 15, 2017 00:06 UTC