Gene Therapy Cures Diabetes in Large Animals

Rebecca Lewis February 08, 2013

After their experiment on mice, researchers from Universitat Autònoma de Barcelona (UAB) in Spain performed a single session of gene therapy on dogs with diabetes. The treatment resulted to healthier animals without any symptom of the disease.

The estimated number of diabetic people around the world is 285 million in 2010, and this figure is expected to increase by 438 million by the end of 2030. Diabetes is among the leading preventable diseases that often result to serious complications, from rapid weight loss to kidney failure, heart disease and amputation. Unfortunately, there is no precise form of treatment for this debilitating condition.

But it seems like there will be – soon. The treatment, which scientists considered ‘less invasive’ involved a single session of various injections in the rear legs using simple needles commonly used in cosmetic procedures. The needles carry the gene therapy vectors (called adeno-associated vectors) which express the insulin gene and the glucokinase – an enzyme that regulates the uptake of glucose from the blood.

Gene therapy has been used to treat different illnesses, but this is the first time that it was proven to work for diabetes.

Safe and Effective Treatment

The gene therapy performed on the animals produced positive effects without any risk. Some of the dogs were monitored for four years but they showed no sign of recurrence. The adeno-associated vectors or AAV are not new in the field of medical science. They are currently used to treat a metabolic disease caused by lipoprotein lipase deficiency and triglyceride accumulation in the blood.

Unlike the standard treatments for diabetes such as insulin injections, gene therapy is only performed once. Some diabetic patients who depend on daily insulin injections still develop severe complications and often experience no improvement in their health and wellbeing. On the other hand, gene therapy provides a long-term relief (as long as four years) from the symptoms of diabetes.

The study paves way to the clinical translation of gene therapy to diabetic animals, and eventually, to human patients.

Source of this article:

UAB researchers cure type 1 diabetes in dogs