Abstract
The twitcher mouse is an authentic murine model of a genetic demyelinating disease globoid cell leukodystrophy. Allogeneic bone marrow transplantation (BMT) in twitcher mice resulted in the clinicopathological improvement. Thus, using green fluorescent protein (GFP) transgenic mice as the donor, we investigated the behavior and fate of the donor cells and the possibility of transdifferentiation of the donor cells into neuroglial cells in the chimeric twitcher mice. GFP + cells were found throughout the brain, most conspicuous in the areas of demyelination. The donor GFP + cells expressed RCA-1, a marker for microglia/macrophages but were never exceed 70% of the entire population of the RCA-1 + microglia/macrophages. There was no convincing evidence that GFP + donor cells expressed markers for neurons, astrocytes, or oligodendrocytes. We concluded BMT is therapeutic for this model. However, this effect is not mediated by donor cells transdifferentiation in the brain.
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