Gene therapy used to cure rodents with diabetes

LONDON, England (Reuters) -- Scientists said Wednesday they have used a new type of gene therapy to cure diabetes in mice and rats that could pave the way for new treatments for millions of people with the disease.

Diabetes is a chronic disease that begins in childhood or early adulthood. Sufferers produce little or no insulin, a hormone that regulates blood sugar levels, and must rely on a strict diet or intravenous injection to control the disorder.

But researchers at Yonsei University in Korea and the University of Calgary in Canada have developed a technique to deliver an altered human insulin gene into mice and rats suffering from type one diabetes.

"This new gene therapy may have potential therapeutic value for the cure of autoimmune diabetes in humans," Ji-Won Yoon, of the University of Calgary, and his colleagues said in a report in the science journal Nature.

People with type one diabetes produce no insulin because their body destroys the insulin-secreting beta cells in the pancreas.

The scientists delivered the altered insulin gene into the diabetic rodents with a modified virus injected into the animals.

After the animals were treated, the altered gene kept the animals' blood sugar at normal levels during the eight-month study.

Although the results are encouraging, Jerrold Olefsky of the University of California in San Diego said it is still a huge leap from treating animals to humans.

"Rodents are quite different from humans with respect to maintaining glucose (sugar) levels, and extending these results to human physiology may prove a challenge," he said in a commentary in Nature.

One of the biggest challenges would be getting the insulin levels right because secretions of the hormone change with diet, age, weight and growth status.

"So a gene-therapy approach must be able to modulate insulin delivery in response to changing needs over time," he said.

"Despite these issues, the paper represents a good example of how basic research can be applied to problems of clinical significance," he added.

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American Diabetes Association
Diabetes Action Research & Education Foundation
Juvenile Diabetes Foundation (JDF) International

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