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