Gene Therapy Corrects Sickle Cell Disease In St. Jude Children’s Research Hospital Laboratory Study

ScienceDaily (Dec. 4, 2008) — Using a harmless virus to insert a corrective gene into mouse blood cells, scientists at St. Jude Children’s Research Hospital have alleviated sickle cell disease pathology. In their studies, the researchers found that the treated mice showed essentially no difference from normal mice. Although the scientists caution that applying the gene therapy to humans presents significant technical obstacles, they believe that the new therapy will become an important treatment for the disease.