The development of genome-editing technology could lead to breakthrough gene therapy. Genome editing has made it possible to easily knock out or modify a target gene, while current gene therapy using a virus vector or plasmid hampering modification with respect to gene replacement therapies. Clinical development using these genome-editing tools is progressing rapidly. However, it is also becoming clear that there is a possibility of unintended gene sequence modification or deletion, or the insertion of undesired genes, or the selection of cells with abnormalities in the cancer suppressor gene p53; these unwanted actions are not possible with current gene therapy. The Science Board of the Pharmaceuticals and Medical Devices Agency of Japan has compiled a report on the expected aspects of such genome-editing technology and the risks associated with it. This article summarizes the history of that discussion and compares the key concepts with information provided by other regulatory authorities.

中文翻译：

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在日本使用当前基因组编辑技术的基因治疗产品方面。

基因组编辑技术的发展可能会导致基因治疗的突破。基因组编辑使得轻松敲除或修改目标基因成为可能，而当前使用病毒载体或质粒的基因疗法阻碍了基因替代疗法的修改。使用这些基因组编辑工具的临床开发进展迅速。然而，人们也越来越清楚，有可能发生非预期的基因序列修改或缺失，或插入不需要的基因，或选择具有抑癌基因p53异常的细胞。; 使用当前的基因疗法，这些不良行为是不可能的。日本制药和医疗器械机构的科学委员会编制了一份关于此类基因组编辑技术的预期方面及其相关风险的报告。本文总结了该讨论的历史，并将关键概念与其他监管机构提供的信息进行了比较。