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The research team and the cured dogs  (Source: Pierre Caufap)
Only one session of injections cured the dogs for over four years

Researchers from Barcelona have developed a new gene therapy that significantly improved the blood glucose levels of dogs with type 1 diabetes -- and could potentially be a cure.

The team was made up of researchers from the Universitat Autònoma de Barcelona, and was led by Fàtima Bosch.

The new gene therapy consists of a single session of multiple injections. These injections presented adeno-associated vectors (AAV) that both expressed the insulin gene, and also glucokinase (an enzyme that regulates an uptake of glucose from the blood). When both genes were expressed, the excess of blood sugar in type 1 diabetic dogs was controlled over a long-term period.

The adeno-associated vectors were derived from non-pathogenic viruses, and simply transferred the two genes to the muscle of the dogs once injected.

This gene therapy was tested on dogs, who received a single administration of the gene therapy. Results showed that the dogs' blood glucose levels were controlled all the time, whether they were eating or fasting. The dogs didn't have any episodes of hypoglycemia, either, which is when blood glucose levels drop too low.

The dogs in the study maintained normal blood glucose levels for more than four years after the injections without requiring any more sessions, and still show no signs of the disease returning. They didn't experience any secondary complications associated with type 1 diabetes in that time period, either.

Since AAV vectors have been previously introduced into skeletal muscle treatments, getting this gene therapy into a clinical setting shouldn't be too difficult. Once this has been accomplished for type 1 diabetic companion animals, it should move on to humans.


Source: Science Daily



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gene therapy
By daboom06 on 2/11/2013 2:57:26 PM , Rating: 2
gene therapy seems too good to be true. i've always had a hard time believing that you could ever produce long-term effects with it, since you have to modify the genetic code of EVERY cell that needs to be edited. and while playing genetics in bacteria is one thing, to convince eukaryotes to pass on the transfected vectors is a whole other matter.

tldr: mammalian genetics just has to be too complicated to edit after embryogenesis.




RE: gene therapy
By geddarkstorm on 2/11/2013 3:35:45 PM , Rating: 2
Thankfully in this case you only need to hit a specific sub-population of cells (and their associated stem cells if necessary); so that makes things quite a bit simpler on that end.

On the other hand, one of the always present concerns with using a virus vector, especially one as adaptable as the adenovirus, is it could mutate to be infectious again (or pick up infectibility by infecting a cell at the same time as another virus does so, which is similar to how influenza can combine swine and human flu strains together to make a monster bug).

On the other hand, I don't think I'd mind too much to have a strain of the common cold that went around handing out sniffles and curing diabetes.


RE: gene therapy
By lagomorpha on 2/11/2013 10:47:15 PM , Rating: 2
quote:
On the other hand, I don't think I'd mind too much to have a strain of the common cold that went around handing out sniffles and curing diabetes.


Would probably make it difficult to get patients to pay for the cure.


RE: gene therapy
By arazok on 2/12/2013 12:11:49 PM , Rating: 2
Unless you also make cold medications.


RE: gene therapy
By sleepeeg3 on 2/11/2013 11:37:28 PM , Rating: 2
Viral vectors can cause cancer if they insert at a gene that regulates the cell cycle and tumor proliferation. That's what killed gene therapy about 15 years back.


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