In what can only be considered a remarkable medical breakthrough, researchers at Universitat Autònoma de Barcelona (UAB) have completely cured type 1 diabetes in dogs after they were injected during a single gene therapy session. The injected gene therapy vectors ensure a healthy expression of glucose, thus the regular insulin shots and associated side effects with the disease are no longer required.
The therapy consists of a single session of minimally invasive injections to the dog’s rear leg with gene therapy vectors, known as adeno-associated vectors (AAV). These vectors, derived from non-pathogenic viruses, are widely used in gene therapy and have been successful in treating several diseases. In the treated dogs, two genes are targeted to the muscle of adult animals – insulin and glucokinase genes. The latter is an enzyme that regulates the uptake of glucose from the blood, and is typically the one that can cause hyperglycemia (excess of blood sugar associated) in diabetes patients.
When the two genes act simultaneously, they work as a glucose sensor automatically regulating the glucose uptake to healthy levels. Multiple clinical trials have been from which it was observed that the diseased dogs recovered their health and no longer showed symptoms of the disease. In one case, one dog was monitored for four years after therapy and still didn’t show any signs of the disease returning.
While human trials might still be a long way, this sounding success in large animals gives hope that type 1 diabetes may be cured for human patients as well using the same therapy.
The study was led by the head of the UAB’s Centre for Animal Biotechnology and Gene Therapy (CBATEG) Fàtima Bosch, and involved the Department of Biochemistry and Molecular Biology of the UAB, the Department of Medicine and Animal Surgery of the UAB, the Faculty of Veterinary Science of the UAB, the Department of Animal Health and Anatomy of the UAB, the Spanish Biomedical Research Centre in Diabetes and Associated Metabolic Disorders (CIBERDEM), the Children’s Hospital of Philadelphia (USA) and the Howard Hughes Medical Institute of Philadelphia (USA).