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Drug Discov Today. 2019 Apr;24(4):992-999. doi: 10.1016/j.drudis.2019.01.007. Epub 2019 Jan 18.

Induced pluripotent stem cells for neural drug discovery.

Author information

1
National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, MD, USA.
2
National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA.
3
University of Alabama at Birmingham, Birmingham, AL, USA.
4
Retrophin, Inc., San Diego, CA, USA.
5
National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, MD, USA. Electronic address: wzheng@mail.nih.gov.

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.

PMID:
30664937
PMCID:
PMC6476685
[Available on 2020-04-01]
DOI:
10.1016/j.drudis.2019.01.007
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