Review

Stem and progenitor cell–based therapy of the human central nervous system

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Abstract

Multipotent neural stem cells, capable of giving rise to both neurons and glia, line the cerebral ventricles of all adult animals, including humans. In addition, distinct populations of nominally glial progenitor cells, which also have the capacity to generate several cell types, are dispersed throughout the subcortical white matter and cortex. A number of approaches have evolved for using neural progenitor cells in cell therapy. Four strategies are especially attractive for clinical translation: first, transplantation of oligodendrocyte progenitor cells as a means of treating the disorders of myelin; second, transplantation of phenotypically restricted neuronal progenitor cells to treat diseases of discrete loss of a single neuronal phenotype, such as Parkinson disease; third, implantation of mixed progenitor pools to treat diseases characterized by the loss of several discrete phenotypes, such as spinal cord injury; and fourth, mobilization of endogenous neural progenitor cells to restore neurons lost as a result of neurodegenerative diseases, in particular Huntington disease. Together, these may present the most compelling strategies and near-term disease targets for cell-based neurological therapy.

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Acknowledgements

I thank M. Nedergaard, N. Roy, M. Windrem, F. Sim, A. Benraiss, S. Wang, E. Chmielnicki and S.-R. Cho for their contributions to the studies discussed, and apologize to the many authors whose relevant work I have not been able to cite in this short review. My thanks to F. Sim for expert help in illustration. Supported by the US National Institutes of Health–National Institute of Neurological Disorders and Stroke, the National Multiple Sclerosis Society, the New York Spinal Cord Injury Research Program, The A–T Children's Project and the CNS foundation, Berlex Bioscience and Merck Research Laboratories.

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  1. Division of Cell and Gene Therapy, Departments of Neurology and Neurosurgery, 601 Elmwood Ave., Box 645, University of Rochester Medical Center, Rochester, New York 14642, USA.

    • Steve Goldman

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Competing interests

S.G. receives laboratory support from Merck Research Laboratories and Berlex Bioscience, and is a consultant to both Merck and Q Therapeutics.

Corresponding author

Correspondence to Steve Goldman.