13 February 2013

Diabetes gene therapy: tested on dogs

Gene therapy for diabetes has been successfully tested in Spain

Copper news

Researchers from the Autonomous University of Barcelona have demonstrated for the first time the possibility of curing type 1 diabetes in large animals in one session of gene therapy, EurekAlert reports! (UAB researchers cure type 1 diabetes in dogs).


Snapshot from the site uab.es – VM

A group led by Fatima Bosch published an article in the profile journal Diabetes (Treatment of Diabetes and Long-term Survival Following Insulin and Glucokinase Gene Therapy – VM), which presents data that the symptoms of the disease disappeared in dogs as a result of a single gene therapy procedure. Monitoring of the condition of the animals showed that in some cases the absence of symptoms lasted for four years.

As the researchers note, "the therapy is minimally invasive." During one session, a gene therapy drug with two genes is injected into the thigh of a dog using the finest needles used in cosmetology. One gene is responsible for the synthesis of insulin, the other for glucokinase, an enzyme that regulates the absorption of glucose from the blood in the liver. The simultaneous functioning of two genes acts as a "glucose sensor" that provides automatic regulation of glucose uptake in the liver, which reduces the likelihood of diabetic hyperglycemia, or coma – a disease-induced increase in blood sugar.

According to Fatima Bosch, the long-term effect of gene therapy in large animals is observed for the first time. The same authors have already demonstrated the possibility of gene therapy of type 1 diabetes in mice. The latest results, according to scientists, make it possible to predict the introduction of a gene therapy approach into veterinary practice, and subsequently into a clinic for the treatment of patients with type 1 diabetes.

In dogs that underwent gene therapy, normal blood glucose levels were maintained regardless of whether they were hungry or full. In addition, the animals' body weight has normalized and no diabetic complications have occurred in the four years since the experimental treatment.

An adeno-associated vector of a new generation was used to introduce genes into the animal body, it is a carrier of genetically engineered structures created on the basis of a non-pathogenic adeno-associated virus (AAV). Such a particle can embed its genome into the genome of the host organism, which makes it attractive as a source material for the creation of gene therapy drugs.

Observations of dogs that received gene injections on an adeno-associated vector indicate the safety of such a carrier.

One of the gene therapy drugs created using an adeno–associated vector - Glybera - for the treatment of a rare disease of fat metabolism, was recently approved by the European Medical Agency and released on the market.

Portal "Eternal youth" http://vechnayamolodost.ru13.02.2013

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