A team of Spanish scientists – from the Biomedicine Institute of the University of Barcelona and the Network Biomedical Research Center for the Physiopathology of Obesity and Nutrition of the Carlos III Health Institute of Madrid – has managed to develop a gene therapy strategy that has been successful with obesity in laboratory mice. , lowering cholesterol and diabetes.
The research, published in the journal Metabolic Engineering, highlights that “the increase in obesity and associated metabolic morbidity is a global health and social problem”, which “requires new therapeutic approaches urgently”. In this sense, gene therapy opens up a whole range of possibilities.
These methods use one or more genes to treat, prevent, or cure a disease or medical disorder. Gene therapy often works by adding new copies of a damaged gene, or by replacing a defective or missing gene in a patient’s cells with a healthy version of that gene. Gene therapy has been used to treat inherited or acquired genetic diseases, such as leukemia.
In this case, the scientific group focused on adipose tissue to treat obesity. Thus, he developed an ex vivo therapy. This means they derived cell transformation from tissue biopsies of the mice. In this, the transformer cells were implanted and then the tissue was transplanted into mice.
“Since it takes place outside the patient’s body, in this case in mice, this type of therapy is much easier to carry out and allows greater control over infected cells”, explains Professor of Biochemistry and Physiology Dr. explains Laura Herrero, who led the study.
“We implanted stem cells derived from adipose tissue subcutaneously and differentiated them into adipocytes in obese mice, in such a way that they expressed a permanently active form of the key enzyme in lipid oxidation: CPT1AM. decrease, hepatic steatosis and allowed cholesterol and glucose levels”, explains the researcher.
Thus, the scientists concluded that transplanting adipocytes (a cell type that stores lipids as an energy reserve) expressing the mitochondrial enzyme CPT1AM reduced obesity and glucose intolerance in mice.
According to Herrero, adipose tissue plays an important role in regulating energy balance and mesenchymal stem cells. For its part, “carnitine palmitoyltransferase 1A (CPT1A) is the enzyme that regulates mitochondrial oxidation of fatty acids. Our aim was to generate adipocytes that express a constitutively active form of CPT1A (CPT1AM) that has been able to improve the obese metabolic phenotype of mice in their implantation”, explained the researcher.
“Mice dosed with this enzyme showed reduced body weight, hepatic steatosis, and serum levels of insulin and cholesterol, along with improved glucose tolerance,” sums up Herrero, who believes the results will be useful for future clinical use of this approach. support the use. Gene therapy to reduce the rate of obesity and cholesterol.