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Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease

Authors
 Jean-Pyo Lee  ;  Mylvaganam Jeyakumar  ;  Evan Y Snyder  ;  Frances M Platt  ;  Thomas N Seyfried  ;  Stuart A Lipton  ;  David Wenger  ;  Gang Tong  ;  Philip Schwartz  ;  Raymond A Dwek  ;  Terry D Butters  ;  Kook In Park  ;  Franz-Josef Muller  ;  Jennifer Meerloo  ;  Scott McKercher  ;  Jonathan Clarke  ;  Gerald Fu  ;  Heather Rose  ;  Dan Clark  ;  Rena C Baek  ;  Pei-Jen Lee  ;  Hiroto Takahashi  ;  Rodolfo Gonzalez 
Citation
 NATURE MEDICINE, Vol.13(4) : 439-447, 2007 
Journal Title
NATURE MEDICINE
ISSN
 1078-8956 
Issue Date
2007
Abstract
Intracranial transplantation of neural stem cells (NSCs) delayed disease onset, preserved motor function, reduced pathology and prolonged survival in a mouse model of Sandhoff disease, a lethal gangliosidosis. Although donor-derived neurons were electrophysiologically active within chimeric regions, the small degree of neuronal replacement alone could not account for the improvement. NSCs also increased brain beta-hexosaminidase levels, reduced ganglioside storage and diminished activated microgliosis. Additionally, when oral glycosphingolipid biosynthesis inhibitors (beta-hexosaminidase substrate inhibitors) were combined with NSC transplantation, substantial synergy resulted. Efficacy extended to human NSCs, both to those isolated directly from the central nervous system (CNS) and to those derived secondarily from embryonic stem cells. Appreciating that NSCs exhibit a broad repertoire of potentially therapeutic actions, of which neuronal replacement is but one, may help in formulating rational multimodal strategies for the treatment of neurodegenerative diseases.
Full Text
http://www.nature.com/nm/journal/v13/n4/full/nm1548.html
DOI
10.1038/nm1548
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Pediatrics (소아과학교실) > 1. Journal Papers
Yonsei Authors
Park, Kook In(박국인) ORCID logo https://orcid.org/0000-0001-8499-9293
URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/97173
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