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Trials of stem cell transplantation in the transgenic SOD1 ALS mice



Trials of stem cell transplantation in the transgenic SOD1 ALS mice



Society for Neuroscience Abstract Viewer & Itinerary Planner : Abstract No 495 15



Death of motor neurons causes weakness, respiratory paralysis and death in amyotrophic lateral sclerosis (ALS). Since it can potentially replace lost motor neurons or enhance motor neuron viability, cell-based therapy is being explored in ALS. This approach is strengthened by the recent observation that a relatively small sub-population (20%) of wild-type cells can rescue motor neuron cell death in chimeric SOD1WT-SOD1G93A mice. In a recent study, transplantation of neuronal progenitor hNT cells (human teratocarcinonma cell-line Ntera 2/D1) into the spinal cord of SOD1G93A transgenic ALS mice increased survival and improved motor function. In several studies, we have investigated the effect of human stem cells on the disease onset and survival of SOD1G93A transgenic ALS mice, following either systemic intravenous or intraspinal transplantation. Using human fetal neural stem cells, we observe a delay in disease onset (13 +/-5.7 days, p=0.0003) and increase in survival (16 days +/-6.0 days, p<0.0001) compared to control cell treated mice following intravenous administration of cells, and a survival benefit of 9 days +/-7.0 days after multi-site intraparenchymal delivery. Immunohistochemical analyses of tissue from treated ALS mice identify transplanted cells in the spinal cord at the terminal stage of disease.

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