Researchers use genetic therapy to cure type 1 diabetes in mice

A team of Baylor College of Medicine researchers may have identified a genetic therapy that can stimulate the growth of new insulin-producing pancreatic cells and reverse the symptoms of type 1 diabetes, according to a recent presentation at the Annual Meeting of the Endocrine Society.

In testing on mice, the investigation team showed that it is possible to stimulate the activity of the neurogenin3 gene, which instructs newly formed pancreatic cells to differentiate into insulin-producing beta cells.

This solves one of the greatest challenges in treating type 1 diabetes. Individuals with the condition generally do not have enough beta cells, therefore they constantly have dangerously low levels of insulin.

However, creating more beta cells alone does not cure the condition. The reason why type 1 diabetics do not have adequate supplies of beta cells is usually because their body's own immune system attacks and destroys these cells. To combat this problem, the researchers turned to further genetic treatment.

Their mouse model showed that adding a gene called CD274 inhibits the activity of the immune system's T cells, which are the units that often attack the beta cells of the pancreas.

By combining therapies that involve the CD274 and neurogenin3 genes, the researchers showed that it is possible to regrow beta cells and prevent their destruction by the immune system in mice. The animal trials showed an 80 percent success rate.

"With just one injection of this gene therapy, the mice remain diabetes-free long term and have a return of normal insulin levels in the body," said Vijay Yechoor, MD, the researcher of the study.

The next step in the development of the therapy would be to apply it to humans, something that is sometimes easier said than done. Proving the effectiveness of a treatment in mice is generally a good first step in creating a new medication, but it does not necessarily mean that the treatment method will apply to people.

However, if further testing proves successful, Yechoor said that the genetic therapy has the potential to be a "curative" treatment for type 1 diabetes.