Generating Corrected Beta Cells from Patients with Genetic Forms of Diabetes
Principal Investigator: James Wells, PhD
Key Personnel: Lawrence Dolan, MD
Diabetes is characterized by the failure to regulate sugar metabolism and can result in blindness, loss of limb and death. Diabetes in children is largely caused by a failure to produce sufficient amounts of the hormone insulin. Genetic forms of diabetes are fairly common in the pediatric population and we believe that there is an undiscovered group of patients for which the genetic causes of diabetes are unknown. Genetic mutations that cause diabetes often do so by disrupting the formation or function of the cells that make insulin, the beta cells. However, if it were possible to correct the genetic defect and grow healthy beta cells they could be used therapeutically to replace the dysfunctional beta cells. Induced pluripotent stem cells appear to be an ideal source for cell replacement therapies because they can be derived from patients and used to generate therapeutic cells for transplantation. This proposal aims to identify patients with genetic forms of diabetes, make stem cell lines with a corrected gene, and generate healthy insulin-producing beta cells that in the future would be used in the treatment of patients with diabetes.