Nanovehicle-based Small Interfering RNA (siRNA) Delivery for Therapeutic Purposes: A New Molecular Approach in Pharmacogenomics.

IF 3.2 Q2 Pharmacology, Toxicology and Pharmaceutics
Javad Akhtari, Alireza Tafazoli, Hassan Mehrad-Majd, Abdolkarim Mahrooz
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引用次数: 2

Abstract

Background: RNA interference (RNAi) is a process for regulating the gene expression in which small interfering RNAs (siRNAs) silence target genes. siRNA-based therapy as a new molecular treatment approach, offers therapeutic prospects for many common diseases such as cancer and cardiovascular disorders. Nevertheless, the efficacy of siRNA delivery has, so far, remained a challenging issue. This is due to their easy degradation through the circulation system and the difficulties in the intracellular delivery to specific tissues where they silence the target genes. There have been many efforts to develop suitable, safe and effective siRNA delivery systems in the past decades. These efforts specifically aimed to protect siRNA from serum nucleases and deliver it to an intracellular region in the desired target cells. In this context, one of the new and popular approaches is nanovehicle-mediated siRNA delivery systems.

Objective: Here, the authors reviewed and highlighted the recent advances in this exciting and fast growing field to help in the development of effective therapeutic tools in controlling human diseases.

Methods: A literature search was conducted from electronic databases such as Pubmed and Google scholar including original articles and review articles.

Conclusion: siRNA delivery systems potentially may be used in future medicine, particularly for untreatable or poorly treated diseases. As we learn more about these delivery systems, we can better use the tremendous opportunities provided by siRNA-based therapeutics. The results of ongoing clinical trials will play an important role in determining whether siRNA-based drugs can be considered as a new class of drugs.

基于纳米载体的小干扰RNA (siRNA)递送用于治疗目的:药物基因组学的新分子方法。
背景:RNA干扰(RNA interference, RNAi)是一种通过小干扰RNA (sirna)沉默靶基因来调控基因表达的过程。sirna治疗作为一种新型的分子治疗手段,为癌症、心血管疾病等常见疾病的治疗提供了广阔的前景。然而,到目前为止,siRNA递送的有效性仍然是一个具有挑战性的问题。这是因为它们很容易通过循环系统降解,而且很难在细胞内递送到特定组织,从而使目标基因沉默。在过去的几十年里,人们一直在努力开发合适、安全、有效的siRNA递送系统。这些努力特别旨在保护siRNA免受血清核酸酶的侵害,并将其传递到所需靶细胞的细胞内区域。在这种情况下,一种新的和流行的方法是纳米载体介导的siRNA递送系统。目的:在此,作者回顾并强调了这一令人兴奋和快速发展的领域的最新进展,以帮助开发有效的治疗工具来控制人类疾病。方法:在Pubmed、Google scholar等电子数据库中检索文献,包括原创文章和综述文章。结论:siRNA递送系统有可能在未来的医学中使用,特别是对于无法治疗或治疗不良的疾病。随着我们对这些输送系统的了解越来越多,我们可以更好地利用基于sirna的治疗方法提供的巨大机会。正在进行的临床试验结果将在确定基于sirna的药物是否可以被视为一类新的药物方面发挥重要作用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Current clinical pharmacology
Current clinical pharmacology PHARMACOLOGY & PHARMACY-
CiteScore
3.60
自引率
0.00%
发文量
0
期刊介绍: Current Clinical Pharmacology publishes frontier reviews on all the latest advances in clinical pharmacology. The journal"s aim is to publish the highest quality review articles in the field. Topics covered include: pharmacokinetics; therapeutic trials; adverse drug reactions; drug interactions; drug metabolism; pharmacoepidemiology; and drug development. The journal is essential reading for all researchers in clinical pharmacology.
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