Journal of Applied Genetics

, Volume 48, Issue 3, pp 269–272 | Cite as

Neural stem cell implantation extends life in Niemann-Pick C1 mice

  • Iram Ahmad
  • Robert E. Hunter
  • Jonathan D. Flax
  • Evan Y. Snyder
  • Robert P. Erickson
Short Communication


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.


cerebellum disease neurodegeneration neurons neuroprotection Niemann-Pick Purkinje cells stem cells stem cell transplantation type C 


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Copyright information

© Institute of Plant Genetics, Polish Academy of Sciences, Poznan 2007

Authors and Affiliations

  • Iram Ahmad
    • 1
  • Robert E. Hunter
    • 1
  • Jonathan D. Flax
    • 2
  • Evan Y. Snyder
    • 3
  • Robert P. Erickson
    • 1
  1. 1.Department of PediatricsUniversity of ArizonaTucsonUSA
  2. 2.Department of NeurologyUniversity of ArizonaTucsonUSA
  3. 3.Burnham Institute for Medical ResearchLa JollaUSA

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