Stem cells in animal models of Huntington disease: A systematic review

Gabriela Delevati Colpo, Erin Furr Stimming, Antonio Lucio Teixeira

Research output: Contribution to journalReview articlepeer-review

17 Scopus citations


Huntington's disease (HD) is an autosomal-dominant neurodegenerative disorder encoding a mutant form of the huntingtin protein (HTT). HD is pathologically characterized by loss of neurons in the striatum and cortex, which leads to progressive motor dysfunction, cognitive decline and behavioral symptoms. Stem cell-based therapy has emerged as a feasible therapeutic approach for the treatment of neurodegenerative diseases and may be effective in alleviating and/or halting the pathophysiological mechanisms underlying HD. Several pre-clinical studies have used stem cells in animal models of HD. Here, we performed a systematic review of preclinical studies to estimate the treatment efficacy of stem cells in animal models of HD. Based on our systematic review, treatment with stem cells significantly improves neurological and behavioral outcomes in animal models of HD. Although promising results were found, the design of animal studies, the types of transplanted cells and the route of administration are poorly standardized and this greatly complicates comparative analysis.

Original languageEnglish (US)
Pages (from-to)43-50
Number of pages8
JournalMolecular and Cellular Neuroscience
StatePublished - Mar 2019
Externally publishedYes


  • Huntington's disease
  • Induced pluripotent stem cells
  • Mesenchymal stem cells
  • Neural stem cells
  • Stem cells
  • Therapy

ASJC Scopus subject areas

  • Molecular Biology
  • Cellular and Molecular Neuroscience
  • Cell Biology


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