Diabetes Mellitus: Gene Therapy

• Gene therapy in diabetes mellitus can be defined as transfer of DNA to somatic cells in order 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 non-toxic vectors for gene transfer in pancreatic insulin producing beta-cell. If available, these gene therapy tools could prevent the autoimmune beta-cell destruction in type 1 diabetes by protecting the remaining beta-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 beta-cells. Such cells could conceivably compensate for the lost endogenous beta-cell mass and restore a regulated insulin secretion.

Nyckelord

Diabetes

gene therapy

insulin

beta-cell

islet

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