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.

中文翻译：

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干细胞基因组编辑在药物发现和治疗应用中的最新进展。

从病毒载体介导到基于蛋白质的编辑（包括锌指核酸酶，TALEN和CRISPR / Cas系统）的基因组工程技术已得到显着改善。这些技术促进了药物的发现，并导致了许​​多难治性疾病的潜在治疗方法的发展。他们可以有效地纠正遗传错误；但是，这些技术具有局限性，例如脱靶效应和可能的安全性问题，在人类中使用这些技术时必须加以考虑。为了克服这些限制并加速这些技术的临床实施，已经做出了巨大的努力。在这篇评论中