Neural stem cell implantation extends life in Niemann-Pick C1 mice
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In order to evaluate the phenotypic effects of implanted neural stem cells (NSCs) in the mouse model of Niemann-Pick C (NPC) disease, we injected a well-characterized clone of murine NSCs into the cerebella of neonatalNpc1 − /− and control mice. The implanted cells survived and were abundant in some regions of the cerebellum. Life span was lengthened in NPC mice with the implanted NSCs. However, the rate of weight gain and subsequent weight loss, resulting from neurodegeneration, was not significantly different from un-injected controls. Ataxia was measured by Rota-Rod performance. The overall rate of decline in time on the Rota-Rod was not significantly slowed down. Thus, in this small group of NPC mice, a single administration in the neonatal period of the NSCs (which were not engineered to over-express the missing gene and not directed into the parenchyma) was only partially therapeutic.
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- Neural stem cell implantation extends life in Niemann-Pick C1 mice
Journal of Applied Genetics
Volume 48, Issue 3 , pp 269-272
- Cover Date
- Print ISSN
- Online ISSN
- Additional Links
- Purkinje cells
- stem cells
- stem cell transplantation
- type C
- Author Affiliations
- 1. Department of Pediatrics, University of Arizona, 1501 N. Campbell Avenue, P.O. Box 245073, 85724-5073, Tucson, AZ, USA
- 2. Department of Neurology, University of Arizona, Tucson, USA
- 3. Burnham Institute for Medical Research, La Jolla, USA