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Gene Therapy and Preventing Diabetes

(Ivanhoe Newswire) -- A recent study at the University of Pittsburgh revealed gene therapy could serve as a means of preventing type 1 diabetes in those who are genetically susceptible to the disease.

Type 1 diabetes, formerly known as juvenile diabetes, affects more than 700,000 Americans and can cause health difficulties like blindness, heart disease, kidney failure and nerve damage.

In the study, researchers inserted a gene into non-obese diabetic (NOD) mice that encoded for a protein called cytokine, which is responsible for the transition of immune cells becoming insulin-producing cells. This allowed researchers to prevent hyperglycemia, more commonly known as high blood sugar, in the mice.

The genes were sent via an adeno-associated virus that inserted genes for one of two cytokines, interleukin-4 (IL-4), which was found to prevent hyperglycemia or interleukin-10 (IL-10), which was found to bring about hyperglycemia more quickly.

"Although the exact mechanism is still under investigation, we believe the protection we observed in our study is due to IL-4 stimulating an increase in regulatory T cells, which are known to suppress the activation of the immune system," reports Khaleel Rehman Khaja, Ph.D., senior research associate, University of Pittsburgh School of Medicine.

According to Dr. Khaja, the finding that researchers can successfully insert genes into a live organism and observe their effects on type 1 diabetes was the most significant part of the study.

SOURCE: American Society of Gene Therapy Annual Meeting, Baltimore, May 31-June 4, 2006