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Recent advances in genome editing of stem cells for drug discovery and therapeutic application

Cited 23 time in Web of Science Cited 28 time in Scopus
Issue Date
2020-05
Citation
Pharmacology and Therapeutics, Vol.209, p. 107501
Keywords
Genome editingStem cellsDrug discoveryTherapeutic application
Abstract
Genome engineering technologies right from viral vector-mediated to protein-based editing-which include zinc finger nucleases, TALENs, and CRISPR/Cas systems-have been improved significantly. These technologies have facilitated drug discovery and have resulted in the development of potential curative therapies for many intractable diseases. They can efficiently correct genetic errors; however, these technologies have limitations, such as off-target effects and possible safety issues, which need to be considered when employing these techniques in humans. Significant efforts have been made to overcome these limitations and to accelerate the clinical implementation of these technologies. In this review, we focus on the recent technological advancements in genome engineering and their applications in stem cells to enable efficient discovery of drugs and treatment of intractable diseases. (C) 2020 The Authors. Published by Elsevier Inc.
ISSN
0163-7258
URI
https://hdl.handle.net/10371/171741
DOI
https://doi.org/10.1016/j.pharmthera.2020.107501
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