Activation of sirtuin 1 as therapy for the peroxisomal disease adrenoleukodystrophy

Cell Death Differ. 2015 Nov;22(11):1742-53. doi: 10.1038/cdd.2015.20. Epub 2015 Mar 27.

Abstract

Oxidative stress and mitochondrial failure are prominent factors in the axonal degeneration process. In this study, we demonstrate that sirtuin 1 (SIRT1), a key regulator of the mitochondrial function, is impaired in the axonopathy and peroxisomal disease X-linked adrenoleukodystrophy (X-ALD). We have restored SIRT1 activity using a dual strategy of resveratrol treatment or by the moderate transgenic overexpression of SIRT1 in a X-ALD mouse model. Both strategies normalized redox homeostasis, mitochondrial respiration, bioenergetic failure, axonal degeneration and associated locomotor disabilities in the X-ALD mice. These results indicate that the reactivation of SIRT1 may be a valuable strategy to treat X-ALD and other axonopathies in which the control of redox and energetic homeostasis is impaired.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adrenoleukodystrophy / drug therapy*
  • Adrenoleukodystrophy / genetics
  • Adrenoleukodystrophy / metabolism
  • Adrenoleukodystrophy / therapy*
  • Animals
  • Blotting, Western
  • Disease Models, Animal
  • Humans
  • In Vitro Techniques
  • Locomotion / drug effects
  • Locomotion / genetics
  • Mice
  • Mice, Mutant Strains
  • Oxidation-Reduction
  • Real-Time Polymerase Chain Reaction
  • Resveratrol
  • Sirtuin 1 / genetics
  • Sirtuin 1 / metabolism*
  • Stilbenes / therapeutic use*

Substances

  • Stilbenes
  • Sirtuin 1
  • Resveratrol