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Research progress on siRNA delivery with nonviral carriers

机译:非病毒载体传递siRNA的研究进展

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摘要

RNA interference is a powerful method for the knockdown of pathologically relevant genes. Small interfering RNAs (siRNAs) have been widely demonstrated as effective biomedical genetic-therapy applications for many diseases. Unfortunately, siRNA duplexes are not ideal drug-like molecules. Problems hindering their effective application fundamentally lie in their delivery, stability, and off-target effects. Delivery systems provide solutions to many of the challenges facing siRNA therapeutics. Due to some fatal disadvantages of viral vectors, nonviral carriers have been studied extensively. Aside from liposomes, nanoparticles and cationic polymer carriers have exhibited improved in vivo stability, better biocompatibility, and efficiency for gene silencing with less cellular toxicity. They may represent a promising strategy for siRNA-based therapies, especially as nanomaterials. The present review also summarizes other methods of siRNA delivery and the side effects of the nanoparticles.
机译:RNA干扰是敲除病理相关基因的有力方法。小干扰RNA(siRNA)已被广泛证明是对许多疾病有效的生物医学遗传疗法应用。不幸的是,siRNA双链体不是理想的药物样分子。阻碍其有效应用的问题从根本上在于其交付,稳定性和脱靶效应。递送系统为siRNA治疗剂面临的许多挑战提供了解决方案。由于病毒载体的某些致命缺点,非病毒载体已被广泛研究。除脂质体外,纳米颗粒和阳离子聚合物载体还显示出改善的体内稳定性,更好的生物相容性以及基因沉默的效率,且细胞毒性较小。它们可能代表基于siRNA的疗法,特别是作为纳米材料的有前途的策略。本综述还总结了siRNA递送的其他方法和纳米颗粒的副作用。

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