Grant: Project Grant

Ubiquinone, also known as Co-enzyme Q10 (CoQ10) is a natural compound found in almost all cells of all organisms, including people. It is absolutely needed for cells to function normally, in particular to be able to produce energy and to fight oxidative stress from free radicals. Patients with an inborn inability to make sufficient amounts of CoQ10 become extremely sick. Furthermore, other types of diseases, for example Parkinson's disease, as well as aging itself, lead to loss of the normal amount of CoQ10. For all these reasons CoQ10 is provided as a treatment in these disease situations. In addition, it is used by many as an anti-aging nutritional supplement. Unfortunately, the chemical properties of CoQ10 are such that it is very difficult for the body and the cells of the body to absorb it when it is provided in the diet. This is why the positive effects of CoQ10 treatment are often very small or absent, despite the fact that they could in principle be very beneficial. We have been able to develop methods to find new chemical compounds that could allow for an increase in the content of CoQ10 in the body and the cells of patients who could benefit from it. We expect to find two types of chemical compounds that could be used as drugs: 1) Compounds that allow for much better absorption of CoQ10 when it is provided as a supplement, and 2) compounds that boost the natural production of CoQ10 in cells. We will test our compounds in a special type of mice that are engineered to have low CoQ10 levels, which makes them very sick, as well as in mutant mice that are sick because of problems with energy production and difficulties with handling (detoxifying) free radicals.