期刊
PHARMACOLOGY & THERAPEUTICS
卷 177, 期 -, 页码 32-43出版社
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.pharmthera.2017.02.026
关键词
Induced pluripotent stem cells; Drug discovery; Disease modeling; Organ-on-chip; Organoid; High throughput screening
资金
- National Health and Medical Research Council [1084256]
- Medical Advances without Animals association fellowship
- Ophthalmic Research Institute of Australia
- Childhood Cancer Trust
- University of Melbourne (Louisa Jean De Bretteville Bequest)
- University of Melbourne (Therapeutic Technologies Research Initiative)
- National Health and Medical Research Council of Australia [1084256] Funding Source: NHMRC
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. (C) 2017 Elsevier Inc. All rights reserved.
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