Induced pluripotent stem cells for neural drug discovery

  • Atena Farkhondeh
  • , Rong Li
  • , Kirill Gorshkov
  • , Kevin G. Chen
  • , Matthew Might
  • , Steven Rodems
  • , Donald C. Lo
  • , Wei Zheng

Research output: Contribution to journalReview articlepeer-review

Abstract

Neurological diseases such as Alzheimer's disease and Parkinson's disease are growing problems, as average life expectancy is increasing globally. Drug discovery for neurological disease remains a major challenge. Poor understanding of disease pathophysiology and incomplete representation of human disease in animal models hinder therapeutic drug development. Recent advances with induced pluripotent stem cells (iPSCs) have enabled modeling of human diseases with patient-derived neural cells. Utilizing iPSC-derived neurons advances compound screening and evaluation of drug efficacy. These cells have the genetic backgrounds of patients that more precisely model disease-specific pathophysiology and phenotypes. Neural cells derived from iPSCs can be produced in a large quantity. Therefore, application of iPSC-derived human neurons is a new direction for neuronal drug discovery.

Original languageEnglish
Pages (from-to)992-999
Number of pages8
JournalDrug Discovery Today
Volume24
Issue number4
DOIs
StatePublished - Apr 2019
Externally publishedYes

ASJC Scopus Subject Areas

  • Pharmacology
  • Drug Discovery

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