Cutting-Edge Gene Therapy Could Cure Hemophilia A


Scientists are one step closer to developing a cure for hemophilia A – the most common type of the blood disorder – after a groundbreaking clinical trial led by the National Health Service (NHS) and conducted by researchers from the Barts Health NHS Trust and Queen Mary University of London.

Hemophilia A is a hereditary genetic disorder that primarily affects men. People with the condition have virtually no blood protein factor VIII, which is necessary for normal blood clotting. Hemophilia A raises the risk for excessive bleeding from the slightest injury, progressive joint damage from bleeding into the joints, and spontaneous internal bleeding, which may be life-threatening. Current treatment involves multiple weekly injections to control and prevent bleeding. There is no cure.

Sourced from: The New England Journal of Medicine