Zatsepin Timofei S, Kotelevtsev Yuri V, Koteliansky Victor
Center of Functional Genomics, Skolkovo Institute of Science and Technology; Department of Chemistry, Lomonosov Moscow State University; Production Department, Central Research Institute of Epidemiology, Moscow, Russia.
Center of Functional Genomics, Skolkovo Institute of Science and Technology.
Int J Nanomedicine. 2016 Jul 5;11:3077-86. doi: 10.2147/IJN.S106625. eCollection 2016.
This review covers the basic aspects of small interfering RNA delivery by lipid nano-particles (LNPs) and elaborates on the current status of clinical trials for these systems. We briefly describe the roles of all LNP components and possible strategies for their improvement. We also focus on the current clinical trials using LNP-formulated RNA and the possible outcomes for therapy in the near future. Also, we present a critical analysis of selected clinical trials that reveals the common logic behind target selection. We address this review to a wide audience, especially to medical doctors who are interested in the application of RNA interference-based treatment platforms. We anticipate that this review may spark interest in this particular audience and generate new ideas in target selection for the disorders they are dealing with.
本综述涵盖了脂质纳米颗粒（LNP）递送小干扰RNA的基本方面，并阐述了这些系统的临床试验现状。我们简要描述了所有LNP组分的作用及其可能的改进策略。我们还重点关注了目前使用LNP配制RNA的临床试验以及近期治疗可能取得的结果。此外，我们对选定的临床试验进行了批判性分析，揭示了靶点选择背后的共同逻辑。我们将本综述面向广大读者，特别是对基于RNA干扰的治疗平台应用感兴趣的医生。我们预计，本综述可能会激发这一特定读者群体的兴趣，并为他们所治疗的疾病的靶点选择产生新的思路。
