Targeted genome editing is an advanced technique that enables precise modification of the nucleic acid sequences in a genome. Genome editing is typically performed using tools, such as molecular scissors, to cut a defined location in a specific gene. Genome editing has impacted various fields of biotechnology, such as agriculture; biopharmaceutical production; studies on the structure, regulation, and function of the genome; and the creation of transgenic organisms and cell lines. Although genome editing is used frequently, it has several limitations. Here, we provide an overview of well-studied genome-editing nucleases, including single-stranded oligodeoxynucleotides (ssODNs), transcription activator-like effector nucleases (TALENs), zinc-finger nucleases (ZFNs), and CRISPR-Cas9 RNA-guided nucleases (CRISPR-Cas9). To this end, we describe the progress toward editable nuclease-based therapies and discuss the minimization of off-target mutagenesis. Future prospects of this challenging scientific field are also discussed.

靶向基因组编辑是一种先进技术，可以精确修饰基因组中的核酸序列。基因组编辑通常使用分子剪刀等工具来切割特定基因中的指定位置。基因组编辑影响了生物技术的各个领域，例如农业；生物制药生产；研究基因组的结构、调控和功能；以及转基因生物和细胞系的创建。尽管基因组编辑经常使用，但它有一些局限性。在这里，我们概述了经过充分研究的基因组编辑核酸酶，包括单链寡脱氧核苷酸 (ssODN)、转录激活子样效应核酸酶 (TALEN)、锌指核酸酶 (ZFN) 和 CRISPR-Cas9 RNA 引导核酸酶（CRISPR-Cas9）。为此，我们描述了基于可编辑核酸酶的疗法的进展，并讨论了脱靶诱变的最小化。还讨论了这个具有挑战性的科学领域的未来前景。