Grant: Project Grant

In persons with type 1 diabetes, the insulin-producing beta cells of the pancreas are destroyed by one's own immune system. Insulin remains the best treatment, but even with close glucose monitoring and insulin administration, persons with diabetes still face complications. A cure for diabetes may lie in the replacement of insulin-producing cells by transplantation. Hundreds of patients worldwide have received transplants of islets enabling better blood glucose control without the need for insulin administration. Yet because there are not enough organ donors, new sources of insulin-producing cells are needed for the millions living with this disease. Moreover, most islet transplants fail within a few years, requiring patients to return to insulin injections. Two recent advances may transform cell therapy in diabetes. First, human insulin-producing cells can now be generated from stem cells in a laboratory dish within a few weeks. Second, the genes of cells can be engineered to produce proteins that may enhance their survival and function. Here will edit the genome of human embryonic stem cells in order to make insulin-producing cells that will function better and survive longer upon transplantation into persons with type 1 diabetes. We will add genes that will protect them from stress, inflammation and adaptive immunity. These modified cells will first be tested by transplantation into mouse models of human diabetes. We are well-poised to move these cells into clinical trial, as our team includes leaders in cell manufacturing and clinical cell therapy. Our goal is to produce a new and improved cell source for cell replacement therapy in diabetes. Such an advance could not only transform the lives of thousands of Canadians living with diabetes but also greatly reduce the tremendous economic and health burden that diabetes places on Canada today.