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

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Title
Recent advances in genome editing of stem cells for drug discovery and therapeutic application
Author(s)
Jaesuk Lee; Delger Bayarsaikhan; Govigerel Bayarsaikhan; Jin-Soo Kim; Elizabeth Schwarzbach; Bonghee Lee
Subject
Drug discovery, ; Genome editing, ; Stem cells, ; Therapeutic application
Publication Date
2020-05
Journal
PHARMACOLOGY & THERAPEUTICS, v.209, pp.107501
Publisher
PERGAMON-ELSEVIER SCIENCE LTD
Abstract
© 2020 The Authors. 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
URI
https://pr.ibs.re.kr/handle/8788114/8631
DOI
10.1016/j.pharmthera.2020.107501
ISSN
0163-7258
Appears in Collections:
Center for Genome Engineering(유전체 교정 연구단) > 1. Journal Papers (저널논문)
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