基于 RNA 干扰的治疗及其递药系统。
RNA interference-based therapy and its delivery systems.
机构信息
Department of Gynecologic Oncology and Reproductive Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Department of Obstetrics and Gynecology, The Second Affiliated Hospital of Harbin Medical University, Harbin, China.
出版信息
Cancer Metastasis Rev. 2018 Mar;37(1):107-124. doi: 10.1007/s10555-017-9717-6.
RNA interference (RNAi) is considered a highly specific approach for gene silencing and holds tremendous potential for treatment of various pathologic conditions such as cardiovascular diseases, viral infections, and cancer. Although gene silencing approaches such as RNAi are widely used in preclinical models, the clinical application of RNAi is challenging primarily because of the difficulty in achieving successful systemic delivery. Effective delivery systems are essential to enable the full therapeutic potential of RNAi. An ideal nanocarrier not only addresses the challenges of delivering naked siRNA/miRNA, including its chemically unstable features, extracellular and intracellular barriers, and innate immune stimulation, but also offers "smart" targeted delivery. Over the past decade, great efforts have been undertaken to develop RNAi delivery systems that overcome these obstacles. This review presents an update on current progress in the therapeutic application of RNAi with a focus on cancer therapy and strategies for optimizing delivery systems, such as lipid-based nanoparticles.
RNA 干扰 (RNAi) 被认为是一种高度特异性的基因沉默方法，在治疗心血管疾病、病毒感染和癌症等各种病理状况方面具有巨大的潜力。尽管 RNAi 等基因沉默方法在临床前模型中得到了广泛应用，但 RNAi 的临床应用具有挑战性，主要是因为难以实现成功的系统递送。有效的递送系统对于实现 RNAi 的全部治疗潜力至关重要。理想的纳米载体不仅解决了递送裸露的 siRNA/miRNA 的挑战，包括其化学不稳定特性、细胞外和细胞内屏障以及先天免疫刺激，还提供了“智能”靶向递送。在过去的十年中，人们已经做出了巨大的努力来开发克服这些障碍的 RNAi 递送系统。本文综述了 RNAi 在癌症治疗等治疗应用方面的最新进展，重点介绍了优化递送系统的策略，如基于脂质的纳米粒子。
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