寡核苷酸药物遗传毒性评价策略的研究进展

doi:10.3969/j.issn.1004-616x.2026.02.012

摘要/Abstract

摘要： 寡核苷酸药物为一类短链核苷酸序列药物，可通过调控基因表达或干扰特定RNA功能发挥靶向治疗作用。因其具备特异性高、靶向能力强的特点，在基因调控、疾病治疗和药物递送等方面展现出巨大潜力。寡核苷酸药物与基因组的序列特异性相互作用可能引发非靶基因杂交，导致转录调控异常，因其化学修饰和作用机制存在特殊性，传统遗传毒性评价方法面临挑战。因此，本文对寡核苷酸药物遗传毒性的潜在作用机制、分级评价策略和组合试验方法及相关监管要求进行了综述。

关键词: 寡核苷酸, 遗传毒性, 安全性评价, 靶向治疗

中图分类号:

R965

蒋晨晨, 黄瑛, 宋捷, 文海若. 寡核苷酸药物遗传毒性评价策略的研究进展[J]. 癌变·畸变·突变, 2026, 38(2): 160-165.

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