New gene therapy strategy cures Type 1 diabetes in mice

A team of researchers at Baylor University were able to cure Type 1 diabetes in mice by tweaking an earlier therapeutic approach that had been defeated by their immune system.

The study co-author, Lawrence Chan, MD, DSc, chief of Baylor's diabetes, endocrinology and metabolism division, had already managed to develop a gene therapy that stimulated the development of new beta cells in the liver, restoring insulin production and normal blood sugar levels in more than 100 mice with chemically induced diabetes. But in non-obese diabetic mice, they had found that the treatment failed after the mouse's immune system killed the newly formed beta cells.

The team combined the gene therapy with interleukin-10 and then injected the therapy intravenously. During more than 20 months of follow-up, the treatment completely reversed Type 1 diabetes in half of the mice. While the therapy did not reverse autoimmunity throughout the body, it protected the new beta cells from the "local destructive effect of autoimmunity."

"We developed a protective 'moat' around the new beta cells," said Chan. "We are now developing other strategies to try to fortify the newly formed beta cells and give them better weapons in addition to the moat, in order to increase the treatment's cure rate."

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