siRNA: Mechanism of action, challenges, and therapeutic approaches
siRNA: Mechanism of action, challenges, and therapeutic approaches
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DOI：
10.1016/j.ejphar.2021.174178
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发表时间：
2021-05-31
影响因子：
5
通讯作者：
Awidi, Abdalla
中科院分区：
文献类型：
--
作者：
Alshaer, Walhan;Zureigat, Hadil;Awidi, Abdalla
由于具有特异性和引人注目的基因沉默作用，RNA 干扰 (RNAi) 有望成为治疗各种感染、血液肿瘤、心血管和神经退行性疾病的重要方法。小干扰RNA (siRNA) 的作用机制基于转录后基因沉默。 siRNA 分子通常特异性且有效地敲低疾病相关基因。然而，它们的特点是细胞摄取低，并且容易受到核酸酶介导的降解的影响。因此，siRNA 需要载体来保护其并有效递送至靶细胞中。当前的综述重点介绍了基于 siRNA 的作用机制、挑战以及临床应用的最新进展。
Owing to specific and compelling gene silencing, RNA interference (RNAi) is expected to become an essential approach in treating a variety of infectious, hemato-oncological, cardiovascular, and neurodegenerative conditions. The mechanism of action of small interfering RNA (siRNA) is based on post-transcriptional gene silencing. siRNA molecules are usually specific and efficient in the knockdown of disease-related genes. However, they are characterized by low cellular uptake and are susceptible to nuclease-mediated degradation. Therefore, siRNAs require a carrier for their protection and efficient delivery into target cells. The current review highlights the siRNA-based mechanism of action, challanges, and recent advances in clinical applications.