Induced pluripotent stem cells for modeling of pediatric neurological disorders

Jiho Jang, Zhejiu Quan, Yunjin J. Yum, Hyo Sook Song, Seonyeol Paek, hoonchul kang

Research output: Contribution to journalReview article

6 Citations (Scopus)

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.

Original languageEnglish
Pages (from-to)871-891
Number of pages21
JournalBiotechnology Journal
Volume9
Issue number7
DOIs
Publication statusPublished - 2014 Jan 1

Fingerprint

Induced Pluripotent Stem Cells
Nervous System Diseases
Pediatrics
Angelman Syndrome
Adrenoleukodystrophy
Friedreich Ataxia
Rett Syndrome
Technology
Prader-Willi Syndrome
Spinal Muscular Atrophy
Fragile X Syndrome
Gene Transfer Techniques
Cell- and Tissue-Based Therapy
Down Syndrome
Neurodegenerative Diseases
Epilepsy
Mutation
Genes
Therapeutics

All Science Journal Classification (ASJC) codes

  • Applied Microbiology and Biotechnology
  • Molecular Medicine

Cite this

Jang, Jiho ; Quan, Zhejiu ; Yum, Yunjin J. ; Song, Hyo Sook ; Paek, Seonyeol ; kang, hoonchul. / Induced pluripotent stem cells for modeling of pediatric neurological disorders. In: Biotechnology Journal. 2014 ; Vol. 9, No. 7. pp. 871-891.
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Induced pluripotent stem cells for modeling of pediatric neurological disorders. / Jang, Jiho; Quan, Zhejiu; Yum, Yunjin J.; Song, Hyo Sook; Paek, Seonyeol; kang, hoonchul.

In: Biotechnology Journal, Vol. 9, No. 7, 01.01.2014, p. 871-891.

Research output: Contribution to journalReview article

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AB - 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.

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