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[IP] Gene therapy developments...

Thursday December 7 5:22 PM ET
Animal Study Shows Gene Therapy May Help Diabetics 

NEW YORK (Reuters Health) - Early research results in mice suggest it may be 
possible to treat diabetes with gene therapy, possibly ridding patients of 
the need to take daily injections of insulin to control their disease.

A team of Canadian and US scientists was able to genetically engineer gut 
cells known as K cells to produce human insulin in mice. This success means 
that researchers could theoretically use gene therapy to overcome the 
fundamental dysfunction behind diabetes in humans, according to a report in 
the December 8th issue of Science.

Diabetes is a chronic disease in which the body either does not produce or 
does not properly use insulin, a hormone that helps control blood sugar, or 
glucose. Normally, pancreas cells called beta cells release insulin in 
response to food intake, moving glucose from the blood to body cells to be 
used for energy.

With type 1 diabetes, however, the immune system attacks beta cells, leaving 
these diabetics with no insulin-producing cells. They must inject synthetic 
insulin every day. People with type 2 diabetes, the form linked to obesity, 
are unable to properly use insulin. Many of these diabetes patients have to 
inject insulin or take it in pill form.

In the current study, researchers looked at whether they could use genes to 
bestow insulin-producing powers upon K cells, which are situated in the 
stomach and small intestine.

A team led by Timothy J. Kieffer of the University of Alberta in Edmonton, 
Canada, introduced a human insulin gene into K cells and then engineered mice 
to carry the new K cells. The investigators found the mice were able to 
produce human insulin. Moreover, the engineered K cells protected the animals 
from developing diabetes even after their insulin-producing beta cells were 

Kieffer told Reuters Health that K cells are ``excellent candidates'' for 
beta cell substitutes in diabetics because K cells already have the 
``necessary machinery'' to store and release insulin.

K cells naturally secrete a hormone called GIP immediately after a meal, 
Kieffer explained. ``Our rationale,'' he said, ``was that if we could 
engineer these K cells to produce insulin, it should be made and stored in 
the cells in advance, ready to be released promptly in response to a meal.''

Kieffer stressed that these findings in mice only show that the concept of 
gene therapy for diabetes is feasible. He said the remaining obstacles 
include figuring out how to get insulin genes into human K cells.

SOURCE: Science 2000;290:1959-1962. 
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