《生命科学》 2025, 37(11): 1411-1420

基因编辑技术医学应用的安全风险与伦理问题

张丽雯¹ , 金成成^1,2 , 刘　晓¹ , 张学博¹ , 朱成姝¹ , 阮梅花¹ , 熊　燕^1,2,*

¹中国科学院上海生命科学信息中心，中国科学院上海营养与健康研究所，上海 200031 ²中国科学院大学，北京 100049

摘　要：

基因编辑技术的快速发展推动着医学领域迈入精准诊疗的新阶段，在遗传疾病治疗、肿瘤干预、传染病防控等领域具有巨大应用潜力。同时，技术的快速迭代与应用场景的不断扩展也引发了诸多安全风险与伦理争议。本文系统梳理了基因编辑技术的发展历程与应用现状，重点分析了该技术在医学应用中的安全风险与伦理问题。安全风险方面，围绕风险识别与风险评估两个维度，结合典型案例揭示脱靶效应、免疫反应及长期影响等风险。伦理问题方面，聚焦医学应用中的伦理挑战，剖析其中的伦理问题与伦理关切点，包括知情同意、隐私保护、社会公平及人类基因资源保护等核心问题。在此基础上，提出构建基于技术迭代的动态风险评估框架、贯穿研发全周期的闭环安全审查体系以及实施基于应用场景的分级分类精准管控建议，以期为基因编辑技术在临床医学实践中的安全、规范与可持续应用提供参考。

通讯作者：熊　燕 , Email:yxiong@sinh.ac.cn

Safety risks and ethical issues in the medical application of gene editing technology

ZHANG Li-Wen¹ , JIN Cheng-Cheng^1,2 , LIU Xiao¹ , ZHANG Xue-Bo¹ , ZHU Cheng-Shu¹ , RUAN Mei-Hua¹ , XIONG Yan^1,2,*

¹Shanghai Information Center for Life Sciences, Shanghai Institute of Nutrition and Health, Chinese Academy of Sciences, Shanghai 200031, China ²University of Chinese Academy of Sciences, Beijing 100049, China

Abstract:

The rapid development of gene editing technologies is propelling the medical field into a new era of recision medicine, offering transformative opportunities for the treatment of genetic diseases, tumor interventions, and infectious disease prevention. However, the accelerated pace of technological iteration and the expansion of clinical applications have generated a range of safety risks and ethical challenges. This study systematically reviews the evolution and current landscape of gene editing technologies, with a particular emphasis on their medical applications. Regarding safety risks, the discussion is structured around the risk identification and assessment, using representative cases to illustrate risks such as off-target effects, immune responses, and long-term impacts. In terms of ethical issues, the article focuses on ethical challenges in medical applications, providing an in-depth analysis of core ethical concerns including informed consent, privacy protection, social equity, and the preservation of human genetic resources. Based on these analyses, the article proposes three main recommendations. First, it proposes constructing a dynamic risk assessment framework that keeps pace with technological advancements. Second, it suggests establishing a closed-loop safety review system to cover the entire research and development lifecycle. Finally, the article recommends implementing scenario-based, graded, and precise regulatory controls. These suggestions aim to provide theoretical support and practical guidance for the safe, standardized, and sustainable clinical application of gene editing technologies.

Communication Author：XIONG Yan , Email:yxiong@sinh.ac.cn