Phenotyping Neurodegeneration in Human iPSCs

Annu Rev Biomed Data Sci. 2021 Jul 20:4:83-100. doi: 10.1146/annurev-biodatasci-092820-025214. Epub 2021 Apr 23.

Abstract

Induced pluripotent stem cell (iPSC) technology holds promise for modeling neurodegenerative diseases. Traditional approaches for disease modeling using animal and cellular models require knowledge of disease mutations. However, many patients with neurodegenerative diseases do not have a known genetic cause. iPSCs offer a way to generate patient-specific models and study pathways of dysfunction in an in vitro setting in order to understand the causes and subtypes of neurodegeneration. Furthermore, iPSC-based models can be used to search for candidate therapeutics using high-throughput screening. Here we review how iPSC-based models are currently being used to further our understanding of neurodegenerative diseases, as well as discuss their challenges and future directions.

Keywords: iPSC; neurodegeneration; omics; phenotype.

Publication types

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

MeSH terms

  • Animals
  • Humans
  • Induced Pluripotent Stem Cells*
  • Neurodegenerative Diseases* / genetics