Modeling Brain Disorders Using Induced Pluripotent Stem Cells

Cold Spring Harb Perspect Biol. 2020 Jun 1;12(6):a035659. doi: 10.1101/cshperspect.a035659.


Brain disorders, from neurodegenerative to psychiatric disorders, are among the most challenging conditions to study because of the intricate nature of the human brain and the limitations of existing model systems in recapitulating all these intricacies. However, innovations in stem cell technologies now allow us to reprogram patient somatic cells to induced pluripotent stem cells (iPSCs), which can then be differentiated to disease-relevant neural and glial cells. iPSCs are a valuable tool to model brain disorders, as they can be derived from patients with known symptom histories, genetics, and drug-response profiles. Here, we discuss the premise and validity of the iPSC-based in vitro model system and highlight key findings from the most commonly studied neurodegenerative and psychiatric disorders.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Brain / physiology
  • Brain / physiopathology*
  • Brain Diseases / metabolism*
  • Cell Differentiation
  • Central Nervous System
  • Chemistry, Pharmaceutical / methods
  • Drug Design
  • Genetic Techniques
  • Humans
  • Induced Pluripotent Stem Cells / metabolism*
  • Models, Biological
  • Models, Neurological
  • Neurodegenerative Diseases
  • Neuroglia / metabolism
  • Neurons / metabolism
  • Pluripotent Stem Cells / physiology
  • Stem Cell Transplantation