Functional nanostructures for effective delivery of small interfering RNA therapeutics.
Functional nanostructures for effective delivery of small interfering RNA therapeutics.
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作者：
Hong CA;Nam YS
小干扰RNA （siRNA）已被证明是通过RNA干扰（RNAi）实现靶特异性基因沉默的有力工具。它控制靶向基因表达的能力给基因治疗作为癌症和遗传疾病的治疗带来了新的希望。然而，siRNA表现出较差的药理学性质，如血清稳定性低、脱靶和先天免疫反应，这给临床应用带来了重大挑战。此外，siRNA由于其阴离子性质和坚硬的结构，不能穿过细胞膜进行RNAi活性。因此，开发一种安全、稳定、高效的系统将siRNA治疗药物输送到靶细胞的细胞质中至关重要。为了克服siRNA治疗应用面临的主要障碍，已经开发了几种用于siRNA递送的纳米颗粒平台。这篇综述涵盖了广泛的非病毒siRNA传递系统，用于增强细胞摄取和体外和体内靶向基因沉默，并讨论了它们的特点和治疗性siRNA的临床应用机会。
Small interfering RNA (siRNA) has proved to be a powerful tool for target-specific gene silencing via RNA interference (RNAi). Its ability to control targeted gene expression gives new hope to gene therapy as a treatment for cancers and genetic diseases. However, siRNA shows poor pharmacological properties, such as low serum stability, off-targeting, and innate immune responses, which present a significant challenge for clinical applications. In addition, siRNA cannot cross the cell membrane for RNAi activity because of its anionic property and stiff structure. Therefore, the development of a safe, stable, and efficient system for the delivery of siRNA therapeutics into the cytoplasm of targeted cells is crucial. Several nanoparticle platforms for siRNA delivery have been developed to overcome the major hurdles facing the therapeutic uses of siRNA. This review covers a broad spectrum of non-viral siRNA delivery systems developed for enhanced cellular uptake and targeted gene silencing in vitro and in vivo and discusses their characteristics and opportunities for clinical applications of therapeutic siRNA.
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