Human glia can both induce and rescue aspects of disease phenotype in Huntington disease

Nat Commun. 2016 Jun 7:7:11758. doi: 10.1038/ncomms11758.

Abstract

The causal contribution of glial pathology to Huntington disease (HD) has not been heavily explored. To define the contribution of glia to HD, we established human HD glial chimeras by neonatally engrafting immunodeficient mice with mutant huntingtin (mHTT)-expressing human glial progenitor cells (hGPCs), derived from either human embryonic stem cells or mHTT-transduced fetal hGPCs. Here we show that mHTT glia can impart disease phenotype to normal mice, since mice engrafted intrastriatally with mHTT hGPCs exhibit worse motor performance than controls, and striatal neurons in mHTT glial chimeras are hyperexcitable. Conversely, normal glia can ameliorate disease phenotype in transgenic HD mice, as striatal transplantation of normal glia rescues aspects of electrophysiological and behavioural phenotype, restores interstitial potassium homeostasis, slows disease progression and extends survival in R6/2 HD mice. These observations suggest a causal role for glia in HD, and further suggest a cell-based strategy for disease amelioration in this disorder.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Behavior, Animal
  • Chimera / metabolism
  • Cognition
  • Crosses, Genetic
  • Disease Progression
  • Female
  • Human Embryonic Stem Cells / metabolism
  • Humans
  • Huntingtin Protein / metabolism
  • Huntington Disease / pathology*
  • Hyaluronan Receptors / metabolism
  • Male
  • Mice
  • Motor Activity
  • Neostriatum / pathology
  • Neuroglia / metabolism
  • Neuroglia / pathology*
  • Neurons / metabolism
  • Phenotype
  • Stem Cell Transplantation
  • Survival Analysis

Substances

  • Htt protein, mouse
  • Huntingtin Protein
  • Hyaluronan Receptors