关键词: Small interfering RNA (siRNA) bioconjugation chemical modification drug delivery gene delivery gene silencing gene therapy nanotechnology targeted gene delivery

Mesh : Humans RNA, Small Interfering RNA Interference Gene Silencing Nanoparticles Genetic Therapy / methods Nanotechnology / methods

来  源:   DOI:10.1080/17460441.2022.2155630

Abstract:
RNA interference (RNAi) using small interfering RNA (siRNA) is a promising strategy to control many genetic disorders by targeting the mRNA of underlying genes and degrade it. However, the delivery of siRNA to targeted organs is highly restricted by several intracellular and extracellular barriers.
This review discusses various design strategies developed to overcome siRNA delivery obstacles. The applied techniques involve chemical modification, bioconjugation to specific ligands, and carrier-mediated strategies. Nanotechnology-based systems like liposomes, niosomes, solid lipid nanoparticles (SLNs), dendrimers, and polymeric nanoparticles (PNs) are also discussed.
Although the mechanism of siRNA as a gene silencer is well-established, only a few products are available as therapeutics. There is a great need to develop and establish siRNA delivery systems that protects siRNAs and delivers them efficiently to the desired sitesare efficient and capable of targeted delivery. Several diseases are reported to be controlled by siRNA at their early stages. However, their targeted delivery is a daunting challenge.
摘要:
未经证实:使用小干扰RNA(siRNA)的RNA干扰(RNAi)是一种有前途的策略,可以通过靶向基础基因的mRNA并降解它来控制许多遗传性疾病。然而,siRNA向靶器官的递送受到几个细胞内和细胞外屏障的高度限制。
UNASSIGNED:这篇综述讨论了几项研究,这些研究报告了克服siRNA递送障碍的各种设计策略。应用的技术包括化学改性,与特定配体的生物缀合,和载体介导的策略。基于纳米技术的系统,如脂质体,Niosomes,固体脂质纳米粒(SLN),树枝状聚合物,和聚合物纳米颗粒(PNs)也进行了讨论。
UNASSIGNED:尽管siRNA作为基因沉默子的机制已经确立,只有少数产品可用作治疗剂。非常需要开发和建立保护siRNA并将其有效递送至所需位点的适当递送系统。几种疾病有望在早期阶段由siRNA控制。然而,他们的目标交付是一个艰巨的挑战。
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