Advances, Nuances, and Potential Pitfalls When Exploiting the Therapeutic Potential of RNA Interference
Advances, Nuances, and Potential Pitfalls When Exploiting the Therapeutic Potential of RNA Interference
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DOI：
10.1002/cpt.8
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发表时间：
2015-01-01
影响因子：
6.7
通讯作者：
Marsden, P. A.
中科院分区：
文献类型：
--
作者：
Battistella, M.;Marsden, P. A.
RNA干扰（RNAi）的发现有可能改变医学治疗的范式。由于能够选择性地沉默基因的功能，RNAi不仅为确定基因的功能提供了不可或缺的研究工具，而且还为开发新的治疗方法提供了潜力，这些治疗方法将抑制与疾病有关的特定基因。通过对RNAi的研究，发现了治疗学的新概念。出现了细微差别。例如，整体RNAi途径可能受到癌症和细胞应激（如缺氧）中的体细胞突变的影响。此外，病毒基因治疗可能对内源性短非编码RNA途径产生意想不到的影响。因此，重要的是要了解RNAi疗法进入加工途径的位置。我们强调了RNAi作为一种新的治疗方法的不断发展，例如淀粉样变性，并解决了与其临床应用相关的一些预期挑战。
The discovery of RNA interference (RNAi) holds the potential to alter the paradigm of medical therapeutics. With the ability to selectively silence the function of a gene, RNAi not only provides an indispensable research tool for determining the function of a gene, but also offers potential for the development of novel therapeutics that will inhibit specific genes involved in disease. New concepts in therapeutics have been uncovered through the study of RNAi. Nuances have emerged. For instance, global RNAi pathways can be affected by somatic mutations in cancer and cellular stress, such as hypoxia. Also, viral gene therapy can have unexpected effects on endogenous short noncoding RNA pathways. Therefore, it is important to understand where RNAi therapeutics enter the processing pathways. We highlight the evolving use of RNAi as a new class of therapeutics, such as for amyloidosis, and address some of the anticipated challenges associated with its clinical application.