Gene Therapy Gives Promising Results In Treating Obesity
U.S. researchers from Ohio State University (USA) showed in mice that gene therapy allowed a sustainable weight loss and improved metabolic parameters of the animal. They want to turn to clinical trials in humans. This research is published in the online scientific journal Nature Medicine, 8 March 2009.

A substance called BDNF (Hypothalamic brain-derived neurotrophic factor), produced in the brain called the hypothalamus, plays a major role in weight regulation. It exists both in animals and humans. The absence of the BDNF gene (which produces the molecule BDNF) in mice makes obese.

Left mouse obese untreated.
Right obese mice treated with gene therapy.
The researchers then injected the gene BDNF directly into the hypothalamus of normal mice, diabetic and subjected to a diet rich in fat. Researchers have also developed a system that can negatively regulate the gene. This system is automatically activated when the mice reached a normal weight.

The gene has been active in the obese mice and progressively as the mice lost weight the expression of BDNF protein was reduced by the automatic activation of negative regulatory system put in place to also wait and stabilize at a normal weight. The researchers then restored a normal regulation of weight, a balance between weight gain and weight loss.

Researchers have found, by observing the mice for the long term, there were no adverse side effects on bone density, the circadian rhythm or the general behavior of mice. The diabetic mice saw their health, their parameters and their physical condition improved.

This method has shown very encouraging results in mice, researchers have made an application to the FDA (U.S. regulatory authority of the drug) to conduct clinical trials in humans. They think they can start them within a year.
Article: Molecular Therapy of obesity and diabetes by a physiological autoregulatory Approach
Authors: Lei Cao, En-Ju D Lin, C Michael Cahill, Chuansong Wang Xianglan Liu & Matthew J During
Journal publication: Nature Medicine

Credit Nature.


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