University of Southern California

Developing a personalized approach to curing genetic forms of diabetes

Senta Georgia (Photo by Cristy Lytal)

Senta Georgia (Photo by Cristy Lytal)

Patients with enteric anendocrinosis are born lacking the gastrointestinal cells that produce hormones. During childhood, they also lose insulin-producing pancreatic cells, known as beta cells, and become severely diabetic.

To better understand this disease, USC Stem Cell researcher Senta Georgia and her colleagues will focus their studies on a young patient who has enteric anendocrinosis due to a mutation in a gene called Neurogenin3 (NEUROG3).

Using the Chang Stem Cell Engineering Facility, genome editing manager Chang Tong and his researchers reprogrammed the patient’s skin cells into induced pluripotent stem cells (iPSCs). They then applied genome editing to correct the NEUROG3 mutation.

In hopes that it will improve the beta cells’ survival and function, Dr. Georgia’s team is using these corrected iPSCs to generate beta cells with the patient’s unique genetic background. If these edited beta cells continue to thrive and produce insulin when transplanted into a diabetic mouse, this will be the first step toward developing a cell replacement therapy for this patient.

Funded by the California Institute of Regenerative Medicine (CIRM), this study has the potential to create a precedent for generating new insulin cells for patients with genetic forms of diabetes. In the future, her team expects to use these cells to replace this patient’s missing gastrointestinal hormone-producing cells.