In a study to be published in the January 2006 issue of Nature Biotechnology, researchers led by a team of scientists at Memorial Sloan-Kettering Cancer Center have devised a novel strategy that uses stem cell-based gene therapy and RNA interference to genetically reverse sickle cell disease (SCD) in human cells. This research is the first to demonstrate a way to genetically correct this debilitating blood disease using RNA interference technology. To prevent the production of the abnormal hemoglobin that causes sickle cell disease, a viral vector was introduced in cell cultures of patients who have the disease. The vector carried a therapeutic globin gene harboring an embedded small interfering RNA precursor designed to suppress abnormal hemoglobin formation. Tested in adult stem cells from SCD patients, researchers found that the newly formed red blood cells made normal hemoglobin and suppressed production of the sickle shaped hemoglobin typical of the disease.
