한빛사 논문
Jaesuk Leea,b,c1, Delger Bayarsaikhana,1, Govigerel Bayarsaikhana,1, Jin-Soo Kimb,c,*, Elizabeth Schwarzbachd,* Bonghee Leea,*
aLee Gil Ya Cancer and Diabetes Institute, Gachon University, Incheon, Republic of Korea
bDepartment of Chemistry, Seoul National University, Seoul, Republic of Korea
cCenter for Genome Engineering, Institute for Basic Science, Daejeon, Republic of Korea
dThe New York Stem Cell Foundation Research Institute, New York, NY, United States
*Correspondence
1These authors contributed equally to this work.
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.
Keywords: Genome editing, Stem cells, Drug discovery, Therapeutic application
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