Conflict of interest: nothing to declare.
Stem cell-based therapy for Huntington's disease†
Article first published online: 18 FEB 2013
Copyright © 2012 Wiley Periodicals, Inc.
Journal of Cellular Biochemistry
Volume 114, Issue 4, pages 754–763, April 2013
How to Cite
Maucksch, C., Vazey, E. M., Gordon, R. J. and Connor, B. (2013), Stem cell-based therapy for Huntington's disease. J. Cell. Biochem., 114: 754–763. doi: 10.1002/jcb.24432
- Issue published online: 18 FEB 2013
- Article first published online: 18 FEB 2013
- Accepted manuscript online: 23 OCT 2012 11:58AM EST
- Manuscript Accepted: 15 OCT 2012
- Manuscript Received: 10 OCT 2012
- STEM CELLS;
- HUNTINGTON'S DISEASE;
- RODENT HD MODEL
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. J. Cell. Biochem. 114: 754–763, 2013. © 2012 Wiley Periodicals, Inc.