Gene therapy in type 1 diabetes.

Type 1 diabetes (T1D) is caused by the autoimmune-mediated destruction of insulin-producing beta cells in the pancreas. T1D affects as many as 3 million patients in the United States alone, with 15,000 new cases developing every year (Juvenile Diabetes Research Foundation), and presently there is no cure for T1D. In recent years, there has been a great deal of interest in developing gene therapy approaches to treat T1D. Gene therapy approaches tend to fall into three broad categoriesthose aimed at preventing or curing autoimmunity, those aimed at restoring insulin production through islet transplant or genetically engineered insulin production, and approaches that aim to prevent the morbidity and mortality associated with this complex disease. We review these studies here.