Journal
DRUG DISCOVERY TODAY
Volume 24, Issue 4, Pages 992-999Publisher
ELSEVIER SCI LTD
DOI: 10.1016/j.drudis.2019.01.007
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Funding
- Intramural Research Program of the Therapeutic Development Branch
- National Center for Advancing Translational Sciences
- National Institutes of Health
- Retrophin
- NGLY1.org
- NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES [ZIATR000018] Funding Source: NIH RePORTER
- NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE [ZICNS003008] Funding Source: NIH RePORTER
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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.
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