Type 1 Diabetes –
Gene Therapy prevented Onset of Diabetic Symptoms in Mice Study
June 4th, 2006
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Researchers from the University of Pittsburgh, Pennsylvania were
successful at preventing mice from developing the onset of hyperglycemia. The method for the prevention was by introducing a gene
encoding for cytokine. Cytokine is a protein that arouses and hinders
the production and/or job of the immune cells from making insulin
producing cells. The research was first presented at the American
Society of Gene Therapy Annual Meeting in Baltimore which was May 31st
through June 4th.
There is currently an estimated 700,000 people in the United States that
have Type 1 diabetes. Type 1 diabetes is caused because of an
autoimmune disorder. The body attacks the insulin producing cells found
in the pancreas. Not only is do type 1 diabetes individuals have
chronic hyperglycemia complications include blindness, kidney failure,
heart disease, and nerve damage. Another name for type 1 diabetes is
juvenile diabetes. The disorder usually begins in children however
adults also can develop this condition later in life as well.
The gene delivery method used is called an adeno-associated virus in
order to introduce the genes for two variations of cytokines that the
researchers were testing. The cytokines tested are named interleukin-4
(IL-4) and interleukin-10 (IL-10). The cytokines would be directed at
the insulin-producing beta cells of non-obese diabetic mice. The
results of the introduced gene therapy using IL-4 prevented the onset of
hyperglycemia in these mice. IL-10 however accelerated the onset of
hyperglycemia.
"We know that the prevention of type 1 diabetes requires early
intervention in the autoimmune process directed against beta cells of
the pancreatic islets. 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. However, the most
important aspect of our study is that we've shown it is now possible to
efficiently insert genes into beta cells in a living organism, allowing
us to analyze the effects that different gene products have on the
progression of type 1 diabetes," said the lead author Khaleel Rehman
Khaja, Ph.D., senior research associate, department of molecular
genetics and biochemistry, University of Pittsburgh School of Medicine.
The use of gene therapy may be beneficial for preventing the onset of
type 1 diabetes if they are genetically have a higher risk.
Nicole Wilson
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