Genetic Surgery: Curing Hemophilia

Katherine High, of Children’s Hospital of Philadelphia, explains that Hemophilia is a rare genetic disorder in which the blood doesn’t clot normally. If you have hemophilia, you may bleed for a longer time than others after an injury. You also may bleed inside your body (internally), especially in your knees, ankles, and elbows. This bleeding can damage your organs and tissues and may be life threatening. Dr. High and her team have developed a gene-repair kit that treats the blood-clotting disorder hemophilia in animal models. The technique replaces a defective gene with a corrected version. Watch Dr. High as she reviews the most recent results of gene therapy trials in humans as well as explore the different techniques that are being studied for the treatment. This is an excellent opportunity to learn how basic science leads to exciting breakthroughs.