Caloric restriction blocks neuropathology and motor deficits in Machado-Joseph disease mouse models through SIRT1 pathway

Nat Commun. 2016 May 11:7:11445. doi: 10.1038/ncomms11445.

Abstract

Machado-Joseph disease (MJD) is a neurodegenerative disorder characterized by an abnormal expansion of the CAG triplet in the ATXN3 gene, translating into a polyglutamine tract within the ataxin-3 protein. The available treatments only ameliorate symptomatology and do not block disease progression. In this study we find that caloric restriction dramatically rescues the motor incoordination, imbalance and the associated neuropathology in transgenic MJD mice. We further show that caloric restriction rescues SIRT1 levels in transgenic MJD mice, whereas silencing SIRT1 is sufficient to prevent the beneficial effects on MJD pathology. In addition, the re-establishment of SIRT1 levels in MJD mouse model, through the gene delivery approach, significantly ameliorates neuropathology, reducing neuroinflammation and activating autophagy. Furthermore, the pharmacological activation of SIRT1 with resveratrol significantly reduces motor incoordination of MJD mice. The pharmacological SIRT1 activation could provide important benefits to treat MJD patients.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Ataxin-3 / metabolism
  • Autophagy / drug effects
  • Caloric Restriction*
  • Cell Line, Tumor
  • Cerebellum / metabolism
  • Cerebellum / pathology
  • Disease Models, Animal
  • Gait
  • Inflammation / pathology
  • Machado-Joseph Disease / metabolism*
  • Machado-Joseph Disease / pathology*
  • Mice, Inbred C57BL
  • Mice, Transgenic
  • Motor Activity* / drug effects
  • Mutant Proteins / metabolism
  • Nervous System / drug effects
  • Nervous System / pathology*
  • Neurons / drug effects
  • Neurons / metabolism
  • Neurons / pathology
  • RNA, Messenger / genetics
  • RNA, Messenger / metabolism
  • Resveratrol
  • Sirtuin 1 / genetics
  • Sirtuin 1 / metabolism*
  • Stilbenes / administration & dosage
  • Stilbenes / pharmacology
  • Stilbenes / therapeutic use

Substances

  • Mutant Proteins
  • RNA, Messenger
  • Stilbenes
  • Ataxin-3
  • Sirtuin 1
  • Resveratrol