Researchers from UAB use gene therapy to cure obesity and type 2 diabetes in mice
A research team from the Universitat Autònoma de Barcelona led by Professor Fatima Bosch (partner in the MYOCURE consortium) has managed to cure obesity and type 2 diabetes in mice using gene therapy.
A single administration of an adeno-associated viral vector (AAV) carrying the FGF21 (Fibroblast Growth Factor 21) gene, resulted in genetic manipulation of the liver, adipose tissue or skeletal muscle to continuously produce the FGF21 protein which acts for the maintenance of a correct energy metabolism. By inducing FGF21 production through gene therapy the animal lost weight and decreased insulin resistance.
The results are even more important since the prevalence of type 2 diabetes and obesity is rising around the world which increases the mortality rate and occurrence of cardiovascular and immune diseases, hypertension, arthritis, neurodegenerative disorders and some types of cancer.
AAV-mediated gene therapy has been approved in Europe and the United States for the treatment of several diseases, due to its efficacy and safety profile and is also used in MYOCURE for the development of new therapeutic products for patients suffering from the rare inherited muscle diseases myotubular myopathy (MTM) and Pompe disease.