Gene Therapy to Fight Obesity

A scientific team from the University of Barcelona and the CIBERobn has designed a strategy to fight obesity and diabetes in mice through ex vivo gene therapy which consists of implanting cells that have been manipulated and transformed in order to treat a disease. 

This is the first study to apply the ex vivo gene therapy technique to generate and implant cells that express the CPT1AM protein, an enzyme that plays a decisive role in many metabolic diseases such as obesity.

“In this new therapy, animal models have been implanted subcutaneously stem cells derived from adipose tissue, differentiated into adipocytes, so that they can express an active form of the CPT1AM protein, an enzyme located in the mitochondria that is key in lipid oxidation and is related to metabolic diseases,” says Laura Herrero, a member of the UB Department of Biochemistry and Physiology.

“As a result, in obese mice, it has been possible to reduce weight, fatty liver (hepatic steatosis), cholesterol and glucose levels. In conclusion, the implantation of adipocytes expressing the mitochondrial enzyme CPT1AM helps to reduce obesity and glucose intolerance in mice.”

Adipose tissue plays a key role in regulating energy balance, and adipose-derived mesenchymal stem cells — cells with the ability for self-renewal — have gained interest in cell therapy.

“Specifically, carnitine palmitoyltransferase 1A (CPT1A) is the enzyme that controls mitochondrial fatty acid oxidation. Our aim was to generate adipocytes that could express a constitutively active form of CPT1A — CPT1AM — capable of burning excess fat and improving the obese metabolic phenotype of mice after implantation.”

The results of the new study support the future clinical use of this ex vivo gene therapy approach as a new strategy to reduce obesity and cholesterol rates in the population.

This preclinical study could open the doors to future therapeutic strategies to address the treatment of obesity, which today represents a global health problem.


M Carmen Soler-Vázquez, María del Mar Romero, Marijana Todorcevic, Katia Delgado, Carles Calatayud, Aleyda Benitez -Amaro, Maria Teresa La Chica Lhoëst, Paula Mera, Sebastián Zagmutt, Marianela Bastías-Pérez, Kevin Ibeas, Núria Casals, Joan Carles Escolà-Gil, Vicenta Llorente-Cortés, Antonella Consiglio, Dolors Serra, Laura Herrero. Implantation of CPT1AM-expressing adipocytes reduces obesity and glucose intolerance in mice. Metabolic Engineering, 2023; 77: 256 DOI: 10.1016/j.ymben.2023.04.010

University of Barcelona. (2023, May 30). Researchers design an innovative strategy to fight obesity through gene therapy. ScienceDaily. Retrieved May 30, 2023 from

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