Gene Editing Treatment for Sickle Cell Disease

Gene editing has been used successfully to repair 20 to 40 percent of stem and progenitor cells taken from the peripheral blood of patients with sickle cell disease, according to Rice University bioengineer Gang Bao. Bao, in collaboration with Baylor College of Medicine, Texas Children''s Hospital and Stanford University, is working to find a cure for the hereditary disease. A single DNA mutation causes the body to make sticky, crescent-shaped red blood cells that contain abnormal hemoglobin and can block blood flow in limbs and organs.