Int. J. Mol. Sci. 2014, 15(2), 1719-1745; doi:10.3390/ijms15021719
Review

Use of Genetically Modified Mesenchymal Stem Cells to Treat Neurodegenerative Diseases

1email, 1,2email and 1,3,* email
Received: 2 December 2013; in revised form: 18 December 2013 / Accepted: 14 January 2014 / Published: 23 January 2014
(This article belongs to the Special Issue Pathology and Treatment of Central Nervous System Diseases)
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
Abstract: The transplantation of mesenchymal stem cells (MSCs) for treating neurodegenerative disorders has received growing attention recently because these cells are readily available, easily expanded in culture, and when transplanted, survive for relatively long periods of time. Given that such transplants have been shown to be safe in a variety of applications, in addition to recent findings that MSCs have useful immunomodulatory and chemotactic properties, the use of these cells as vehicles for delivering or producing beneficial proteins for therapeutic purposes has been the focus of several labs. In our lab, the use of genetic modified MSCs to release neurotrophic factors for the treatment of neurodegenerative diseases is of particular interest. Specifically, glial cell-derived neurotrophic factor (GDNF), nerve growth factor (NGF), and brain derived neurotrophic factor (BDNF) have been recognized as therapeutic trophic factors for Parkinson’s, Alzheimer’s and Huntington’s diseases, respectively. The aim of this literature review is to provide insights into: (1) the inherent properties of MSCs as a platform for neurotrophic factor delivery; (2) the molecular tools available for genetic manipulation of MSCs; (3) the rationale for utilizing various neurotrophic factors for particular neurodegenerative diseases; and (4) the clinical challenges of utilizing genetically modified MSCs.
Keywords: mesenchymal stem cells; genetic engineering; Parkinson’s; Alzheimer’s; Huntington’s; neurodegenerative diseases; transplantation; glial cell-derived neurotrophic factor (GDNF); brain derived neurotrophic factor (BDNF); nerve growth factor (NGF)
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MDPI and ACS Style

Wyse, R.D.; Dunbar, G.L.; Rossignol, J. Use of Genetically Modified Mesenchymal Stem Cells to Treat Neurodegenerative Diseases. Int. J. Mol. Sci. 2014, 15, 1719-1745.

AMA Style

Wyse RD, Dunbar GL, Rossignol J. Use of Genetically Modified Mesenchymal Stem Cells to Treat Neurodegenerative Diseases. International Journal of Molecular Sciences. 2014; 15(2):1719-1745.

Chicago/Turabian Style

Wyse, Robert D.; Dunbar, Gary L.; Rossignol, Julien. 2014. "Use of Genetically Modified Mesenchymal Stem Cells to Treat Neurodegenerative Diseases." Int. J. Mol. Sci. 15, no. 2: 1719-1745.

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