Gradalis, Inc., Dallas, TX, USA.
Expert Rev Mol Med. 2010 Aug 18;12:e26. doi: 10.1017/S1462399410001584.
RNA interference (RNAi) is an evolutionary conserved mechanism for specific gene silencing. This mechanism has great potential for use in targeted cancer therapy. Understanding the RNAi mechanism has led to the development of several novel RNAi-based therapeutic approaches currently in the early phases of clinical trials. It remains difficult to effectively deliver the nucleic acids required in vivo to initiate RNAi, and intense effort is under way in developing effective and targeted systemic delivery systems for RNAi. Description of in vivo delivery systems is not the focus of this review. In this review, we cover the rationale for pursuing personalised cancer therapy with RNAi, briefly review the mechanism of each major RNAi therapeutic technique, summarise and sample recent results with animal models applying RNAi for cancer, and provide an update on current clinical trials with RNAi-based therapeutic agents for cancer therapy. RNAi-based cancer therapy is still in its infancy, and there are numerous obstacles and issues that need to be resolved before its application in personalised therapy focusing on patient-cancer-specific targets can become standard cancer treatment, either alone or in combination with other treatments.
RNA 干扰（RNAi）是一种用于特定基因沉默的进化保守机制。该机制在靶向癌症治疗中有很大的应用潜力。对 RNAi 机制的理解导致了几种新型基于 RNAi 的治疗方法的发展，目前处于临床试验的早期阶段。仍然难以有效地将体内起始 RNAi 所需的核酸递送到体内，目前正在努力开发有效的靶向全身 RNAi 递送系统。体内递送系统的描述不是本综述的重点。在本综述中，我们讨论了用 RNAi 进行个体化癌症治疗的基本原理，简要回顾了每种主要 RNAi 治疗技术的机制，总结和采样了最近应用 RNAi 治疗癌症的动物模型的结果，并更新了目前基于 RNAi 的癌症治疗剂的临床试验。基于 RNAi 的癌症治疗仍处于起步阶段，在将其应用于针对患者-癌症特异性靶标的个体化治疗之前，需要解决许多障碍和问题，无论是单独使用还是与其他治疗方法联合使用，都可以成为标准的癌症治疗方法。
