Gene editing may soon change treatments for diabetes. Researchers have used the gene editing tool CRISPR-Cas9 to enhance human skin to form insulin-producing cells that helped cure the disease in the lab.
The new study, published in the journal Science Translational Medicine, shows the health benefits of using stem cells produced from the skin of a patient with a genetic form of insulin-dependent diabetes called Wolfram syndrome. CRISPR enabled researchers to send the cells into patients and correct a genetic defect that caused diabetes.
The team from Washington University School of Medicine in St. Louis said the new approach appears promising to cure diabetes caused by a single gene mutation. But they hope to see the treatment available for patients with more common forms of diabetes, such as type 1 and type 2.
“This is the first time CRISPR has been used to fix a patient’s diabetes-causing genetic defect and successfully reverse diabetes,” Jeffrey Millman, co-senior investigator and an assistant professor at Washington University, said in a statement. “For this study, we used cells from a patient with Wolfram syndrome because, conceptually, we knew it would be easier to correct a defect caused by a single gene. But we see this as a stepping stone toward applying gene therapy to a broader population of patients with diabetes.”
Wolfram syndrome requires people to get insulin injections multiple times each day. The condition has been linked to various health conditions, such as problems with vision and balance, and even early death.
The potential diabetes treatment uses pancreatic beta cells formed from human stem cells. Researchers said the converted cells produce more insulin when they encounter blood sugar.
For the study, the team tested the new approach with mice that developed Wolfram syndrome. The animals that received the beta cells were able to more efficiently produce insulin.
Researchers said the diabetes disappeared quickly and blood sugar levels remained normal for six months after the treatment.
“We basically were able to use these cells to cure the problem, making normal beta cells by correcting this mutation,” Fumihiko Urano, co-senior investigator and a professor at Washington University, said. “It’s a proof of concept demonstrating that correcting gene defects that cause or contribute to diabetes — in this case, in the Wolfram syndrome gene — we can make beta cells that more effectively control blood sugar.”
Researchers hope to improve the potential diabetes treatment with gene editing to help provide “personalized regenerative gene therapy.” Aside from human skin, the team plans to use urine samples from patients to produce stem cells that could correct the disease.
