Scientists Are Hopeful About Gene Therapy to Cure People Who Thought They’d Have Hemophilia Forever
14th August 2018
The gene therapy technique involves inserting a mutated version of the blood-clotting gene into a virus and delivering it to the patient’s body, often through an intravenous drip. But the treatment could be described as a one-hit wonder — if it stops working, scientists have not found a way to successfully administer the therapy a second time.
Global biotech company BioMarin ran a trial for patients with hemophilia A in which most patients reached normal levels of blood-clotting protein in their blood, reported The New York Times Tuesday. The levels dropped after a year but remained high enough to make a positive difference for patients for whom the therapy worked.