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

Abstract:

: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 neonatal Npc1(-/-) 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.

journal_name

J Appl Genet

authors

Ahmad I,Hunter RE,Flax JD,Snyder EY,Erickson RP

doi

10.1007/BF03195222

subject

Has Abstract

pub_date

2007-01-01 00:00:00

pages

269-72

issue

3

eissn

1234-1983

issn

2190-3883

pii

403

journal_volume

48

pub_type

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