"I like to think of it as a truck delivering cargo to a garage." That's the way Dr. Margaret Ragni describes gene therapy. However, she goes into much more detail in a study published last week in the New England Journal of Medicine. Dr. Ragni is a medical professor at Pitt and the Director of the Hemophilia Center of Western Pennsylvania. She was part of a multi-site clinical trial using gene therapy to deliver a functional copy of a blood clotting gene to liver cells. The promising results show a 90 percent reduction in internal bleeding and more durability over traditional factor infusions.
