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Type 1 Diabetes – Treatable with Gene Therapy?

A step forward in gene therapy may mark further benefits for those suffering from Type 1 Diabetes.

By applying gene therapy to dogs for up to four years, minus the effects of hypoglycaemia, the advance could prove advantageous for diabetics. The change from using the methods on mice and using dogs (namely Beagles) instead has shown positive success.

“Moving from mice to large animals is a big step,” said Dr. Fatima Bosch, Director of the Center of Animal Biotechnology and Gene Therapy. “If something works well in large animals, we have reason to believe, based on the previous experience in the field of gene transfer, that it is likely that we will see a similar outcomes in humans. For example, gene therapy that worked well in large animals to treat haemophilia is now seeing positive results in clinical trials with humans. We hope that in a few years, we’ll be able to test this therapy for type 1 diabetes in humans as well.”

The dogs were given insulin and glucokinase through an adenoassociated virus (AAV) vector – a vehicle which transports DNA using a virus that is not designed to harm its host. It can still “infect” them, but the cargo could contain whatever the creator wishes. In this case, the insulin and glocokinase is the cargo, which is beneficial.

Insulin is a very familiar component for those suffering from either type of diabetes, which helps to regulate the glucose in the blood. Whilst naturally created by the pancreas, those suffering from diabetes go through the issue that the pancreas produces too little, meaning that the body’s blood sugar is not regulated properly. Through injections and (or) a healthy diet, this is often maintained quite well. Glucokinase is partnered to insulin, which also helps to regulate the rise and fall of circulating glucose levels.

By injecting them into the dogs, researchers found that the dogs maintained normal blood glucose levels for up to four years without any signs of hypoglycaemia and without any need of further injections.

With further testing proving to be promising, the researchers hope to be able to test on humans in the future.

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