Abstract：
Gene editing ushers in a new era of disease treatment since many genetic diseases are caused by base-pair mutations in genomic DNA. With the rapid development of genome editing technology, novel editing tools such as base editing and prime editing (PE) have attracted public attention, heralding a great leap forward in this field. PE, in particular, is characterized by no need for double-strand breaks (DSBs) or homology sequence templates with variable application scenarios, including point mutations as well as insertions or deletions. With higher editing efficiency and fewer byproducts than traditional editing tools, PE holds great promise as a therapeutic strategy for human diseases. Subsequently, a growing demand for the standard construction of PE system has spawned numerous easy-to-access internet resources and tools for personalized prime editing guide RNA (pegRNA) design and off-target site prediction. In this review, we mainly introduce the innovation and evolutionary strategy of PE systems and the auxiliary tools for PE design and analysis. Additionally, its application and future potential in the clinical field have been summarized and envisaged.
摘要：
基因编辑开启了疾病治疗的新时代，因为许多遗传疾病是由基因组DNA中的碱基对突变引起的。随着基因组编辑技术的快速发展,诸如基础编辑和原始编辑（PE）之类的新颖编辑工具引起了公众的关注，预示着这一领域的巨大飞跃。PE,特别是,其特点是不需要双链断裂（DSB）或具有可变应用场景的同源序列模板，包括点突变以及插入或缺失。与传统编辑工具相比，编辑效率更高，副产品更少，PE作为人类疾病的治疗策略具有很大的前景。随后，对PE系统标准构建的不断增长的需求催生了许多易于访问的互联网资源和工具，用于个性化的原始编辑指导RNA（pegRNA）设计和脱靶位点预测。在这次审查中,主要介绍了PE系统的创新和进化策略以及PE设计和分析的辅助工具。此外,总结并展望了其在临床领域的应用和未来潜力。
