Akhtari Javad, Tafazoli Alireza, Mehrad-Majd Hassan, Mahrooz Abdolkarim
Immunogenetics Research Center, Department of Medical Nanotechnology, School of Advanced Technologies in Medicine, Mazandaran University of Medical Sciences, Sari, Iran.
Medical Genetics Research Center, Mashhad University of Medical Sciences, Mashhad, Iran.
Curr Clin Pharmacol. 2018;13(3):173-182. doi: 10.2174/1574884713666180709152610.
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.
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.
A literature search was conducted from electronic databases such as Pubmed and Google scholar including original articles and review articles.
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.
RNA干扰（RNAi）是一种调节基因表达的过程，其中小干扰RNA（siRNA）使靶基因沉默。基于siRNA的疗法作为一种新的分子治疗方法，为许多常见疾病如癌症和心血管疾病提供了治疗前景。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。这是因为它们容易在循环系统中降解，并且在细胞内递送至它们使靶基因沉默的特定组织时存在困难。在过去几十年中，人们为开发合适、安全且有效的siRNA递送系统做出了许多努力。这些努力特别旨在保护siRNA免受血清核酸酶的影响，并将其递送至所需靶细胞的细胞内区域。在这种情况下，新的且流行的方法之一是纳米载体介导的siRNA递送系统。
在此，作者回顾并强调了这个令人兴奋且快速发展的领域的最新进展，以帮助开发控制人类疾病的有效治疗工具。
从电子数据库如Pubmed和谷歌学术进行文献检索，包括原创文章和综述文章。
siRNA递送系统可能在未来医学中使用，特别是用于无法治疗或治疗效果不佳的疾病。随着我们对这些递送系统了解得更多，我们可以更好地利用基于siRNA的疗法提供的巨大机会。正在进行的临床试验结果将在确定基于siRNA的药物是否可被视为一类新药物方面发挥重要作用。
