【摘要】 近年来基于RNA干扰(RNA interference, RNAi)的基因治疗技术在肿瘤治疗方面引起广泛关注。在常规药物治疗无效的情况下，RNAi为癌症患者带来了新的希望。但是，由于小分子干扰RNA(small interfering RNA,siRNA)在体内存在易降解、难递送等问题，极大地限制了其临床转化潜力。纳米载体以其独特的尺寸效应和多样的修饰策略，能够介导高效、靶向的RNA递送，以实现其基因沉默。本文综述了RNAi在基因治疗中的作用机制以及体内递送siRNA的不同载体，介绍了载体体内递送siRNA的主要障碍和作用靶点，并比较了不同载体在siRNA递送中的优势和不足，为新载体的设计提供借鉴，推动RNA干扰疗法向临床的转化。更多还原

【Abstract】 In recent years, gene therapy based on RNA interference(RNAi) has attracted wide attention in tumor therapy. RNAi offers new hope for cancer patients when conventional drug treatments are ineffective. However, due to the problems such as easy degradation and difficult delivery of small interfering RNA(siRNA) in vivo, its clinical transformation potential is greatly limited. With its unique size effect and various modification strategies, nano-carrier can mediate efficient and targeted RNA delivery to achieve gene silencing. This paper reviews the mechanism of RNAi in gene therapy and different carriers for delivering siRNA in vivo, introduces the main obstacles and targets of siRNA delivery in vivo, and compares the advantages and disadvantages of different carriers in siRNA delivery, so as to provide reference for the design of new vectors and promote the transformation of RNAi therapy to clinic.更多还原