A group of researchers are testing gene therapy as a potential way to prevent the immune system from attacking healthy insulin-producing cells in the pancreas.
There are two types of immune cells important to the development of type 1 diabetes: T-effector cells and T-regulatory cells. T-effector cells attack foreig, disease-causing material, while T-regulatory cells prevent T-effector cells from getting out of hand. The goal of this gene research therapy is to trigger the T-regulatory cells to prevent T-effector cells from attacking insulin-producing beta cells in the pancreas, and thereby prevent the development of type 1 diabetes.
To achieve this, a team of researchers led by Maria Grazia Roncarolo, MD inserted an insulin gene fragmented into the liver of a mouse model of type 1 diabetes. The results were positive: the mouse model stimulated the T-regulatory cells in pre-diabetic mice, preventing the immune system from attacking healthy beta cells. If the therapy was conducted at a very early stage – before any symptoms were noticeable – it prevented the onset of type 1 diabetes.
A similar kind of therapy, one that was specifically aimed at T-effector cells, lowered high blood glucose levels in a group of mice with type 1 diabetes and reversed the disease in 75 per cent of the treated mice.
The research is funded by the JDRF.

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