Gene Therapy for Diabetes

The use of gene transfer technologies has allowed the development of potential therapeutic strategies for the treatment of type 1 diabetes and type 2 diabetes, as well as the performance of proof-of-concept studies in small and large animal models to understand pathophysiology of these diseases better. Gene therapy is a therapeutic strategy based on genetic modification of a cell by introduction of exogenous genetic material to prevent, correct, or ameliorate the symptoms of a disease. The use of viral vectors exploits the natural ability of viruses to enter cells, transfer their genetic material to the nucleus, and express proteins. The immune system is a key component of the aetiopathogenesis of type 1 diabetes. Future studies are required to demonstrate the efficacy and safety in large animal models of the approaches that have demonstrated promise in small animals before any of these gene therapies are brought to the clinic to treat diabetes in humans.