CNS disease models with human pluripotent stem cells in the CRISPR age

Curr Opin Cell Biol. 2016 Dec:43:96-103. doi: 10.1016/j.ceb.2016.10.001. Epub 2016 Oct 19.

Abstract

In vitro differentiation of human pluripotent stem cells provides a systematic platform to investigate the physiological development and function of the human nervous system, as well as the etiology and consequence when these processes go awry. Recent development in three-dimensional (3D) organotypic culture systems allows modeling of the complex structure formation of the human CNS, and the intricate interactions between various resident neuronal and glial cell types. Combined with an ever-expanding genome editing and regulation toolkit such as CRISPR/Cas9, it is now a possibility to study human neurological disease in the relevant molecular, cellular and anatomical context. In this article, we review recent progress in 3D neural culture and the implications for disease modeling.

Publication types

  • Review

MeSH terms

  • Animals
  • Central Nervous System Diseases / metabolism
  • Central Nervous System Diseases / pathology*
  • Clustered Regularly Interspaced Short Palindromic Repeats / genetics*
  • Genetic Engineering
  • Humans
  • Models, Biological*
  • Organoids / pathology
  • Pluripotent Stem Cells / cytology*