Gene therapy for sickle cell disease passes key preclinical test
A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, researchers from Dana-Farber/Boston Children’s Cancer and Blood Disorders Center report today in the Journal of Clinical Investigation. The work sets the stage for bringing a decades-old discovery about sickle cell disease to the bedside. A clinical gene therapy trial, using a virus rendered harmless in the laboratory, is expected to launch in the coming year. Sickle cell disease is caused by a mutation in hemoglobin, the oxygen-carrying protein in red blood cells, which causes the usually pliant cells to stiffen and assume a curved or “sickle” shape. These sickle cells live for less time than those with healthy hemoglobin, leading to anemia. They also bind together, leading to blood-flow blockage that can cause severe pain and organ damage throughout the body. View all posts in Science & Health ...