[iPS cell technologies toward overcoming neurological diseases]

Nihon Yakurigaku Zasshi. 2023;158(1):57-63. doi: 10.1254/fpj.22084.
[Article in Japanese]

Abstract

Neurological diseases are often life threatening, with severely affecting an individual's quality of life. However, the disease mechanisms are still less understood, mainly because of lacking good disease models. Over the past decades, researchers developed many models using cell lines or animals, but most of them did not faithfully recapitulate the disease phenotypes. In particular, it is almost impossible to create animal models for multifactorial diseases or sporadic cases of unknown etiology. In these circumstances, it has come to be expected that induced pluripotent stem cells (iPSCs) can revolutionize neurological disease research as they retain patient's genetic information and provide an expandable source of disease-relevant neurons and glial cells. iPSC technologies are now widely used for disease modeling, and further for drug discovery and regenerative medicine. They are also enabling previously infeasible studies such as those uncovering how disease-associated single nucleotide polymorphism (SNP) and genetic variants increase the disease risk. This review describes a variety of iPSC technologies to produce various types of neurons and brain-like tissues (brain organoids) and summarize recent trends in iPSC technology-based neurological disease research. We also discuss the remaining challenges for understanding and overcoming brain disorders.

Publication types

  • Review
  • English Abstract

MeSH terms

  • Animals
  • Brain
  • Induced Pluripotent Stem Cells*
  • Nervous System Diseases* / therapy
  • Neurons
  • Quality of Life