Gene therapy approach for T1 diabetes could alter immune system to boost insulin

The Juvenile Diabetes Research Foundation has taken a special interest in gene therapy as an approach to nipping Type 1 diabetes in the bud, keeping the body's T-effector cells from attacking insulin-producing beta cells.

A team at Stanford University showed in a diabetic mouse model that they could insert an insulin gene fragment into the liver to stimulate the T-regulatory cells into holding the T-effector cells at bay. So while the T-effector cells aim to attack the insulin-producing beta cells because of faulty genetic wiring, the T-regulatory cells are able to stop them from doing so.

The gene insertion halted the progression of Type 1 diabetes at the early stages of the disease before the symptoms were noticeable. The team, led by Maria Grazia Roncarlo of Stanford, also demonstrated that the gene therapy could reduce high blood glucose levels in mice closer to disease onset, according to a JDRF report, and reversed the disease at later stages in 75% of the mice.

This is an important step forward in realizing the potential of gene therapy as a way to treat diabetes. Drug delivery systems for insulin, like needles or pumps, merely alter the amount of insulin available and can be cumbersome and life-altering. With an early diagnosis, this approach shows that it could be possible to severely knock down the effects of Type 1 diabetes, and in many cases, reverse it entirely.

- here's the JDRF report

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