Induced Pluripotent Stem Cells in Huntington's Disease Research: Progress and Opportunity

J Huntingtons Dis. 2016 Jun 28;5(2):99-131. doi: 10.3233/JHD-160199.


Induced pluripotent stem cells (iPSCs) derived from controls and patients can act as a starting point for in vitro differentiation into human brain cells for discovery of novel targets and treatments for human disease without the same ethical limitations posed by embryonic stem cells. Numerous groups have successfully produced and characterized Huntington's disease (HD) iPSCs with different CAG repeat lengths, including cells from patients with one or two HD alleles. HD iPSCs and the neural cell types derived from them recapitulate some disease phenotypes found in both human patients and animal models. Although these discoveries are encouraging, the use of iPSCs for cutting edge and reproducible research has been limited due to some of the inherent problems with cell lines and the technological differences in the way laboratories use them. The goal of this review is to summarize the current state of the HD iPSC field, and to highlight some of the issues that need to be addressed to maximize their potential as research tools.

Keywords: HTT; Huntingtin; Huntington’s disease; induced neuron; induced pluripotent stem cell; neural stem cell; neurodegenerative disorder.

Publication types

  • Review
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Biomedical Research*
  • Humans
  • Huntingtin Protein / genetics
  • Huntingtin Protein / metabolism*
  • Huntington Disease / genetics
  • Huntington Disease / pathology*
  • Huntington Disease / therapy
  • Induced Pluripotent Stem Cells / physiology*


  • Huntingtin Protein