Towards gene therapy of diabetes mellitus

A definitive treatment for diabetes mellitus will be one that maintains a n ormal blood glucose concentration in the face of fluctuating dietary intake . To accomplish this, there must be mechanisms to sense the amount of blood glucose coupled to rapid release of the right amount of insulin. While mec hanical devices to accomplish this are being developed, ultimately the best approach is likely to be based on genetic modification of cells. These cou ld be pancreatic beta-cells, which are the only cells that produce insulin, or other cells that are involved in the pathogenesis of diabetes. Although definitive treatment of diabetes using genetically modified cells is a lon g-term goal, much progress is being made. This review discusses various app roaches to modifying cells genetically, both in vitro and in vivo, for the treatment of diabetes.