Cell-replacement therapy for Huntington's disease is one of very few therapies that has reported positive outcomes in clinical trials. However, for cell transplantation to be made more readily available, logistical, standardization and ethical issues associated with the current methodology need to be resolved. To achieve these goals, it is imperative that an alternative cell source be identified. One of the key requirements of the cells is that they are capable of acquiring an MSN (medium spiny neuron) morphology, express MSN markers such as DARPP-32 (dopamine- and cAMP-regulated phosphoprotein of 32 kDa), and function in vivo in a manner that replicates those that have been lost to the disease. Developmental biology has progressed in recent years to provide a vast array of information with regard to the key signalling events involved in the proliferation, specification and differentiation of striatal-specific neurons. In the present paper, we review the rationale for cell-replacement therapy in Huntington's disease, discuss some potential donor sources and consider the value of developmental markers in the identification of cells with the potential to develop an MSN phenotype.
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February 2009
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Conference Article|
January 20 2009
Medium spiny neurons for transplantation in Huntington's disease
Claire M. Kelly;
Claire M. Kelly
1
*Brain Repair Group, School of Biosciences, Museum Avenue, Cardiff University, Cardiff CF10 3AX, Wales, U.K.
1To whom correspondence should be addressed (email kellycm@cardiff.ac.uk).
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Stephen B. Dunnett;
Stephen B. Dunnett
*Brain Repair Group, School of Biosciences, Museum Avenue, Cardiff University, Cardiff CF10 3AX, Wales, U.K.
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Anne E. Rosser
Anne E. Rosser
*Brain Repair Group, School of Biosciences, Museum Avenue, Cardiff University, Cardiff CF10 3AX, Wales, U.K.
†Departments of Neurology and Genetics, School of Medicine, Cardiff University, Heath Park, Cardiff CF14 4XN, Wales, U.K.
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Publisher: Portland Press Ltd
Received:
October 03 2008
Online ISSN: 1470-8752
Print ISSN: 0300-5127
© The Authors Journal compilation © 2009 Biochemical Society
2009
Biochem Soc Trans (2009) 37 (1): 323–328.
Article history
Received:
October 03 2008
Citation
Claire M. Kelly, Stephen B. Dunnett, Anne E. Rosser; Medium spiny neurons for transplantation in Huntington's disease. Biochem Soc Trans 1 February 2009; 37 (1): 323–328. doi: https://doi.org/10.1042/BST0370323
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