Diabetes Mellitus: Gene Therapy

Abstract

Gene therapy in diabetes mellitus can be defined as the transfer of deoxyribonucleic acid (DNA) to somatic cells to understand, treat or prevent the disease. For many gene therapy strategies in the treatment of diabetes, successful transduction of insulin‐producing cells is a prerequisite. Therefore, much effort is currently directed in developing efficient and nontoxic vectors for gene transfer in pancreatic insulin‐producing β‐cell. If available, these gene therapy tools could prevent the autoimmune β‐cell destruction in type 1 diabetes by protecting the remaining β‐cell mass in newly diagnosed diabetics or in prediabetic individuals at a high risk of becoming diabetic. Such an approach may also prove useful for promoting islet–graft survival after transplantation in diabetic patients. Alternatively, attempts are being made to genetically engineer cells to become artificial β‐cells. Such cells could conceivably compensate for the lost endogenous β‐cell mass and restore a regulated insulin secretion.