By the way there is an interesting interview at the forefront of this Saturday 1/2/2014
Fà Tima Bosch: "Gene therapy can improve the treatment of diabetes"
Fàtima Bosch, director of the Center for Animal Biotechnology and Gènica Teràpia
When Fàtima Bosch began her first project as director of a research group in 1990, she decided to set an ambitious objective.It was proposed to cure diabetes.A difficult goal, certainly, but it didn't seem impossible.
For 24 years it has tried multiple strategies.Since type 1 diabetes is because the pancreas does not produce insulin, it tried to produce the liver.It was not enough.As with the liver it was not enough, he tried to act on the muscle.It was not enough either.He tried to replace insulin with glycoquinase.Neither...
After more than twenty years alternating joys and disappointments, advancing by trial and error, he has finally put a gene therapy that has successfully treated diabetes in dogs.Its next great objective is to extend therapy to people so that they can control their diabetes without having to inject insulin.
How has you managed to control diabetes in dogs?
We have administered two genes, that of insulin and glycoquinase.Together they act as a sensor that effectively and safely regulates the level of glucose (or sugar) in the blood.
Why do the insulin gene need?
It is the missing hormone in type 1 diabetes makes the glucose that circulates in the blood be captured by the cells to be used as energy.
And that of glycoquinase?
It allows control of the blood glucose level faster and more precise than if we only use insulin.Glucoquinase increases glucose capture by cells after eating.But when the glucose level decreases, glycoquinase becomes inactive.It acts as a security mechanism.
How have the dogs that have received the treatment responded?
We have treated a dozen diabetic Beagle dogs.Gene therapy has worked in all.They were dogs that were very sick and now are fine.They have recovered vitality and have a good control of blood glucose in both fasting and after eating, and both when they are still and when they do intense physical activity.
How is a gene therapy administered?
We have injected the genes into the muscles of the rear legs.We act on the muscle because 70% of the glucose that circulates in the blood after having ingested food is captured by the muscles.
How often do you have to repeat the injections?
It is not necessary to repeat them, only once enough!The first dog we treat received gene therapy more than six years ago and continues to perfectly control its glucose level.
Has he surprised that the effects are so durable?
The truth is not.Think that muscle cells are not divided once the fibers have formed.They last for many years.Therefore, if we introduce genes into these cells, they can stay there acting in the long term.
How does it do it for genes to reach the core of the cells where they should act?
We use what we call viral vectors.These are viruses to which their own genes have been removed and we have introduced the genes of insulin and glycoquinase.Since they have the natural ability to enter the cells.We use them as vehicles.
Can they cause infections?
No, because the original genetic material of the virus is no longer.
And could they cause some kind of cancer?
No, because the genetic material we introduce is not integrated into the DNA of the cell.It is floating in the nucleus.It is a very safe therapy.
What is missing for diabetic people to benefit from this advance?
The next step of researchIt will consist of rehearsing therapy in company dogs.So far we have tried it in experimentation dogs that weigh about ten kilos.From now on we have to administer it in diabetic dogs of up to 70 kilos like Rottweilers.
Couldn't they go directly to people?
No, because we have to establish the dose with the maximum possible precision before moving to people.That is why we need to rehearse the therapy in a dog that weighs more or less as a person.
Wouldn't it be better to start with a low dose in people and in any case increase it later?
I have received many letters from patients offered as volunteers to rehearse gene therapy.But these types of essays can only be approved if there are expectations that existing diabetes treatments will improve.To do this, we must establish the dose before asking for authorization for the essay in people.
Have any pharmaceutical company showed interest in bringing this therapy to patients?
There are several that are interested.Gene therapy can improve the treatment of diabetes, and also other genetic diseases, and in the last twenty months the biopharmaceutical industry has invested 300 million euros in gene therapy.Now there is a huge interest in this research area.
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En 1922 descubrieron la insulina, en 1930 la insulina lenta. ¿Que c*** han hecho desde entonces?