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Drug discovery using induced pluripotent stem cell models of neurodegenerative and ocular diseases


Hung, SSC and Khan, S and Lo, CY and Hewitt, AW and Wong, RCB, Drug discovery using induced pluripotent stem cell models of neurodegenerative and ocular diseases, Pharmacology and Therapeutics, 177 pp. 32-43. ISSN 0163-7258 (2017) [Refereed Article]

Copyright Statement

Copyright 2017 Elsevier Inc.

DOI: doi:10.1016/j.pharmthera.2017.02.026


The revolution of induced pluripotent stem cell (iPSC) technology provides a platform for development of cell therapy, disease modeling and drug discovery. Recent technological advances now allow us to reprogram a patient's somatic cells into induced pluripotent stem cells (iPSCs). Together with methods to differentiate these iPSCs into disease-relevant cell types, we are now able to model disease in vitro using iPSCs. Importantly, this represents a robust in vitro platform using patient-specific cells, providing opportunity for personalized precision medicine. Here we provide a review of advances using iPSC for drug development, and discuss the potential and limitations of iPSCs for drug discovery in neurodegenerative and ocular diseases. Emerging technologies that can facilitate the search for new drugs by assessment using in vitro disease models will also be discussed, including organoid differentiation, organ-on-chip, direct reprogramming and humanized animal models.

Item Details

Item Type:Refereed Article
Keywords:disease modeling, drug discovery, high throughput screening, induced pluripotent stem cells, organ-on-chip, organoid
Research Division:Biomedical and Clinical Sciences
Research Group:Ophthalmology and optometry
Research Field:Ophthalmology
Objective Division:Health
Objective Group:Clinical health
Objective Field:Clinical health not elsewhere classified
UTAS Author:Hewitt, AW (Professor Alex Hewitt)
ID Code:121328
Year Published:2017
Web of Science® Times Cited:28
Deposited By:Menzies Institute for Medical Research
Deposited On:2017-09-23
Last Modified:2022-08-25

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