Abstract
Cell-based therapies such as neural transplantation have, until recently, been reserved for focal or regionally restricted neurologic diseases. These are best exemplified by Parkinson’s disease, in which encouraging progress in the use of neural transplantation, especially the grafting of fetal tissue, has been made experimentally (1,2) and clinically (3). [Even recent clinical studies that seemed to call into question such efficacy indicated that implanted fetal cells do exert a local impact albeit one that seemed to provoke an “overdose” effect (4)]. Donor tissue replaces dopamine via the engraftment and enhanced survival of neurotransmitter-secreting cells within the striatum or by forestalling degeneration of dopaminergic cells within the substantia nigra. However, the pathologic lesions of most neurogenetic diseases—indeed, most neurologic disorders—are usually widely disseminated in the brain and spinal cord and have not typically been regarded as within the purview of neural transplantation. Such diseases include not only the inherited neurodegenerative diseases of the pediatric age group (e.g., the lysosomal storage diseases, the leukodystrophies, inborn errors of metabolism, hypoxic—ischemic encephalopathy) but also such adult maladies as Alzheimer’s disease (AD), Huntington’s disease (HD), multi-infarct dementia, multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS), and brain tumors (especially glioblastomas). Therapeutic approaches for such “global” problems have typically depended on pharmacologic or genetic interventions; they have been regarded as beyond the purview of cellular-mediated approaches. Cell replacement therapies have largely been limited to transplantation of somatic cells derived from the hematopoietic system administered via bone marrow transplantation (BMT). In the majority of these disorders, such strategies have been unsatisfactory for treating the central nervous system (CNS) component of the disease.
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Park, K.I. et al. (2003). Global Gene and Cell Replacement Strategies Via Stem Cells. In: Zigova, T., Snyder, E.Y., Sanberg, P.R. (eds) Neural Stem Cells for Brain and Spinal Cord Repair. Contemporary Neuroscience. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-59259-298-2_11
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