Nucleic acid-based therapy emerges as a powerful weapon for the treatment of tumors thanks to its direct, effective, and lasting therapeutic effect. Encouragingly, continuous nucleic acid-based drugs have been approved by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Despite the tremendous progress, there are few nucleic acid-based drugs for brain tumors in clinic. The most challenging problems lie on the instability of nucleic acids, difficulty in traversing the biological barriers, and the off-target effect. Herein, nucleic acid-based therapy for brain tumor is summarized considering three aspects: (i) the therapeutic nucleic acids and their applications in clinical trials; (ii) the various administration routes for nucleic acid delivery and the respective advantages and drawbacks. (iii) the strategies and carriers for improving stability and targeting ability of nucleic acid drugs. This review provides thorough knowledge for the rational design of nucleic acid-based drugs against brain tumor.

基于核酸的疗法因其直接、有效和持久的治疗效果而成为治疗肿瘤的有力武器。令人鼓舞的是，基于核酸的连续药物已获得美国食品药品监督管理局 (FDA) 和欧洲药品管理局 (EMA) 的批准。尽管取得了巨大进展，但临床上用于脑肿瘤的核酸药物却很少。最具挑战性的问题在于核酸的不稳定性、难以穿越生物屏障以及脱靶效应。在此，从三个方面总结了基于核酸的脑肿瘤治疗：（i）治疗性核酸及其在临床试验中的应用；(ii) 核酸递送的各种给药途径以及各自的优缺点。(iii)提高核酸药物稳定性和靶向性的策略和载体。本综述为合理设计抗脑肿瘤核酸药物提供了全面的知识。