Ubiquinol-10 ameliorates mitochondrial encephalopathy associated with CoQ deficiency

Biochim Biophys Acta. 2014 Jul;1842(7):893-901. doi: 10.1016/j.bbadis.2014.02.008. Epub 2014 Feb 24.


Coenzyme Q10 (CoQ10) deficiency (MIM 607426) causes a mitochondrial syndrome with variability in the clinical presentations. Patients with CoQ10 deficiency show inconsistent responses to oral ubiquinone-10 supplementation, with the highest percentage of unsuccessful results in patients with neurological symptoms (encephalopathy, cerebellar ataxia or multisystemic disease). Failure in the ubiquinone-10 treatment may be the result of its poor absorption and bioavailability, which may be improved by using different pharmacological formulations. In a mouse model (Coq9(X/X)) of mitochondrial encephalopathy due to CoQ deficiency, we have evaluated oral supplementation with water-soluble formulations of reduced (ubiquinol-10) and oxidized (ubiquinone-10) forms of CoQ10. Our results show that CoQ10 was increased in all tissues after supplementation with ubiquinone-10 or ubiquinol-10, with the tissue levels of CoQ10 with ubiquinol-10 being higher than with ubiquinone-10. Moreover, only ubiquinol-10 was able to increase the levels of CoQ10 in mitochondria from cerebrum of Coq9(X/X) mice. Consequently, ubiquinol-10 was more efficient than ubiquinone-10 in increasing the animal body weight and CoQ-dependent respiratory chain complex activities, and reducing the vacuolization, astrogliosis and oxidative damage in diencephalon, septum-striatum and, to a lesser extent, in brainstem. These results suggest that water-soluble formulations of ubiquinol-10 may improve the efficacy of CoQ10 therapy in primary and secondary CoQ10 deficiencies, other mitochondrial diseases and neurodegenerative diseases.

Keywords: CoQ(10) deficiency; Mitochondrial encephalopathy; Mouse model; Ubiquinol-10.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Ataxia / drug therapy*
  • Brain Diseases / drug therapy
  • Brain Stem / drug effects
  • Corpus Striatum / drug effects
  • Electron Transport / drug effects
  • Mice
  • Mice, Inbred C57BL
  • Mitochondria / drug effects*
  • Mitochondrial Diseases / drug therapy*
  • Mitochondrial Encephalomyopathies / drug therapy*
  • Muscle Weakness / drug therapy*
  • Oxidative Stress / drug effects
  • Ubiquinone / analogs & derivatives*
  • Ubiquinone / deficiency*
  • Ubiquinone / pharmacology


  • Ubiquinone
  • ubiquinol-10
  • Ubiquinone Q2

Supplementary concepts

  • Coenzyme Q10 Deficiency
  • Mitochondrial encephalopathy