Insulin injection has been the only treatment option for most of the millions of insulin-dependent diabetic individuals. Whole pancreas transplantation has been a successful approach for some patients. It is a complex operation with many potential complications. Recently, it was demonstrated that a glucocorticoid-free immunosuppressive regimen led to remarkably successful islet transplantation. However, both pancreas and islet cell transplantation have to overcome the shortage of cadaveric pancreases that are available for transplantation. The ultimate goal of diabetes therapy is to generate an unlimited source of cells with glucose-responsive insulin secretion that can be transplanted without the need for systemic immunossuppression. The focus of this review is how gene therapy can be used in various approaches in order to develop such a source of cells. The recent advances in b-cell growth and development will also be discussed.
Gene therapy; Diabetes mellitus; beta-cell