Insulin Gene Therapy for Treating Type I Diabetes Mellitus: History, Progress and Future Challenges

Type 1 Diabetes Mellitus (T1DM) results from the autoimmune destruction of insulin-producing pancreatic β cells. Despite advances in our understanding of both the pathogenesis and management of diabetes and treatment options, which mainly include injectable insulin and whole pancreas transplant, these advancements remain unsatisfactory and only a small portion of patients achieve their glycemic goals. Insulin gene therapy refers to the development of β cell substitutes by introducing an insulin producing gene into endogenous cell populations that do not normally produce insulin. This strategy has the promise to overcome the limitations of current treatments. Here, we present an overview of the history of insulin gene therapy and review current achievements. We also briefly discuss areas for improvement and potential future directions.