A new type of gene therapy can bring about long-term remission of type 1 diabetes in rodents, with no detectable adverse effects, paving the way for clinical trials in humans.
Ji-Won Yoon and his colleagues at Yonsei University, Seoul, Korea, and at the University of Calgary, Canada, report that they can cure rodents of diabetes by engineering them to produce a single-chain analogue of insulin that is active without the need for biological processing in a glucose-sensitive manner [Nature, Vol. 408, No. 6811, 23 November 2000, pp. 483–488]. The team delivers the gene for the insulin by infecting the rodent with a genetically modified virus.
Diabetics produce insufficient insulin because of a malfunction in the beta cells of their pancreas. Insulin is the hormone that regulates blood sugar levels. Normally beta cells rapidly release insulin into the blood in response to changes in blood glucose concentration.
"[This] paper represents a definite step forwards, and offers a good example of how fundamental research can be applied to problems of human health," comments Jerrold M. Olefsky of the University of California at San Diego, La Jolla, in an accompanying News and Views article.
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