Modeling human neurological disorders with induced pluripotent stem cells

Yoichi Imaizumi, Hideyuki Okano

Research output: Contribution to journalReview article

65 Citations (Scopus)

Abstract

Human induced pluripotent stem (iPS) cells obtained by reprogramming technology are a source of great hope, not only in terms of applications in regenerative medicine, such as cell transplantation therapy, but also for modeling human diseases and new drug development. In particular, the production of iPS cells from the somatic cells of patients with intractable diseases and their subsequent differentiation into cells at affected sites (e.g., neurons, cardiomyocytes, hepatocytes, and myocytes) has permitted the in vitro construction of disease models that contain patient-specific genetic information. For example, disease-specific iPS cells have been established from patients with neuropsychiatric disorders, including schizophrenia and autism, as well as from those with neurodegenerative diseases, including Parkinson's disease and Alzheimer's disease. A multi-omics analysis of neural cells originating from patient-derived iPS cells may thus enable investigators to elucidate the pathogenic mechanisms of neurological diseases that have heretofore been unknown. In addition, large-scale screening of chemical libraries with disease-specific iPS cells is currently underway and is expected to lead to new drug discovery. Accordingly, this review outlines the progress made via the use of patient-derived iPS cells toward the modeling of neurological disorders, the testing of existing drugs, and the discovery of new drugs.

Original languageEnglish
Pages (from-to)388-399
Number of pages12
JournalJournal of Neurochemistry
Volume129
Issue number3
DOIs
Publication statusPublished - 2014 May

Keywords

  • Parkinson's disease
  • human disease model
  • induced pluripotent stem cells
  • neurological disorders

ASJC Scopus subject areas

  • Biochemistry
  • Cellular and Molecular Neuroscience

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