iPSC-based disease modeling and drug discovery in cardinal neurodegenerative disorders

Hideyuki Okano, Satoru Morimoto

Research output: Contribution to journalReview articlepeer-review

44 Citations (Scopus)


It has been 15 years since the birth of human induced pluripotent stem cell (iPSC) technology in 2007, and the scope of its application has been expanding. In addition to the development of cell therapies using iPSC-derived cells, pathological analyses using disease-specific iPSCs and clinical trials to confirm the safety and efficacy of drugs developed using iPSCs are progressing. With the innovation of related technologies, iPSC applications are about to enter a new stage. This review outlines advances in iPSC modeling and therapeutic development for cardinal neurodegenerative diseases, such as amyotrophic lateral sclerosis, Parkinson's disease, and Alzheimer's disease.

Original languageEnglish
Pages (from-to)189-208
Number of pages20
JournalCell stem cell
Issue number2
Publication statusPublished - 2022 Feb 3


  • AD
  • ALS
  • Alzheimer's disease
  • PD
  • Parkinson's disease
  • amyotrophic lateral sclerosis
  • disease modeling
  • drug repositioning
  • iPSCs
  • induced pluripotent stem cells
  • stratification

ASJC Scopus subject areas

  • Molecular Medicine
  • Genetics
  • Cell Biology


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