Stem cell-based therapy for Huntington's disease

J Cell Biochem. 2013 Apr;114(4):754-63. doi: 10.1002/jcb.24432.


Huntington's disease (HD) is a late-onset neurodegenerative disease characterized by a progressive loss of medium spiny neurons in the basal ganglia. The development of stem cell-based therapies for HD aims to replace lost neurons and/or to prevent cell death. This review will discuss pre-clinical studies which have utilized stem or progenitor cells for transplantation therapy using HD animal models. In several studies, neural stem and progenitor cells used as allotransplants and xenografts have been shown to be capable of surviving transplantation and differentiating into mature GABAergic neurons, resulting in behavioral improvements. Beneficial effects have also been reported for transplantation of stem cells derived from non-neural tissue, for example, mesenchymal- and adipose-derived stem cells, which have mainly been attributed to their secretion of growth and neurotrophic factors. Finally, we review studies using stem cells genetically engineered to over-express defined neurotrophic factors. While these studies prove the potential of stem cells for transplantation therapy in HD, it also becomes clear that technical and ethical issues regarding the availability of stem cells must be solved before human trials can be conducted.

Publication types

  • Review

MeSH terms

  • Adipose Tissue / cytology
  • Adipose Tissue / metabolism
  • Animals
  • Brain / metabolism
  • Brain / pathology*
  • Brain-Derived Neurotrophic Factor / genetics
  • Brain-Derived Neurotrophic Factor / metabolism
  • Cell Differentiation
  • GABAergic Neurons / metabolism
  • GABAergic Neurons / pathology
  • Humans
  • Huntington Disease / pathology
  • Huntington Disease / therapy*
  • Mice
  • Mice, Transgenic / genetics
  • Mice, Transgenic / metabolism
  • Neural Stem Cells / cytology
  • Neural Stem Cells / metabolism
  • Neural Stem Cells / transplantation
  • Rats
  • Repetitive Sequences, Nucleic Acid
  • Stem Cell Transplantation*


  • Brain-Derived Neurotrophic Factor
  • BDNF protein, human