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Induced pluripotent stem cells for modeling of pediatric neurological disorders

Authors
 Jiho Jang  ;  Zhejiu Quan  ;  Yunjin J. Yum  ;  Hyo Sook Song  ;  Seonyeol Paek  ;  Hoon-Chul Kang 
Citation
 BIOTECHNOLOGY JOURNAL, Vol.9(7) : 871-881, 2014 
Journal Title
BIOTECHNOLOGY JOURNAL
ISSN
 1860-6768 
Issue Date
2014
MeSH
Animals ; Child ; Child Development/physiology ; Disease Models, Animal ; Humans ; Induced Pluripotent Stem Cells/cytology* ; Models, Neurological ; Nervous System Diseases/therapy* ; Neurodegenerative Diseases/therapy*
Keywords
Cell-based therapy ; Disease modeling ; Drug screening ; Induced pluripotent stem cells ; Pediatric neurological disorders
Abstract
The pathophysiological mechanisms underlying childhood neurological disorders have remained obscure due to a lack of suitable disease models reflecting human pathogenesis. Using induced pluripotent stem cell (iPSC) technology, various neurological disorders can now be extensively modeled. Specifically, iPSC technology has aided the study and treatment of early-onset pediatric neurodegenerative diseases such as Rett syndrome, Down syndrome, Angelman syndrome. Prader–Willi syndrome, Friedreich's ataxia, spinal muscular atrophy (SMA), fragile X syndrome, X-linked adrenoleukodystrophy (ALD), and SCN1A gene-related epilepsies. In this paper, we provide an overview of various gene delivery systems for generating iPSCs, the current state of modeling early-onset neurological disorders and the ultimate application of these in vitro models in cell therapy through the correction of disease-specific mutations.
Full Text
http://onlinelibrary.wiley.com/doi/10.1002/biot.201400010/abstract
DOI
10.1002/biot.201400010
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Pediatrics (소아과학교실) > 1. Journal Papers
1. College of Medicine (의과대학) > Dept. of Physiology (생리학교실) > 1. Journal Papers
1. College of Medicine (의과대학) > Yonsei Biomedical Research Center (연세의생명연구원) > 1. Journal Papers
Yonsei Authors
Kang, Hoon Chul(강훈철) ORCID logo https://orcid.org/0000-0002-3659-8847
Paek, Seon Yeol(백선열)
Song, Hyo Sook(송효숙)
Yum, Yunjin J.(염윤진)
Jang, Ji Ho(장지호) ORCID logo https://orcid.org/0000-0001-5551-3514
URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/99841
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