Recent advances in genome sequencing have greatly improved our ability to understand and identify the causes of genetic diseases. However, there remains an urgent need for innovative, safe, and effective treatments for these diseases. CRISPR-based genome editing systems have become important and powerful tools in the laboratory, and efforts are underway to translate these into patient therapies. Therapeutic base editing is one form of genome engineering that has gained much interest because of its simplicity, specificity, and effectiveness. Base editors are a fusion of a partially deactivated Cas9 enzyme with nickase function, together with a base-modifying enzyme. They are capable of precisely targeting and repairing a pathogenic mutation to restore the normal function of a gene, ideally without disturbing the rest of the genome. In the past year, research has identified new safety concerns of base editors and sparked new innovations to improve their safety. In this review, we provide an overview of the recent advances in the safety and effectiveness of therapeutic base editors and prime editing.

中文翻译：

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CRISPR/Cas9 编辑：根据新疗法的需求引发关于安全性的讨论。

基因组测序的最新进展极大地提高了我们理解和识别遗传疾病原因的能力。然而，仍然迫切需要对这些疾病进行创新、安全和有效的治疗。基于 CRISPR 的基因组编辑系统已成为实验室中重要且强大的工具，并且正在努力将其转化为患者疗法。治疗性碱基编辑是基因组工程的一种形式，由于其简单性、特异性和有效性而备受关注。碱基编辑器是具有切口酶功能的部分失活的 Cas9 酶与碱基修饰酶的融合。它们能够精确定位和修复致病突变，以恢复基因的正常功能，理想情况下不会干扰基因组的其余部分。在过去的一年里，研究已经确定了碱基编辑器的新安全问题，并激发了新的创新以提高其安全性。在这篇综述中，我们概述了治疗性碱基编辑和主要编辑的安全性和有效性的最新进展。