基于RNA干扰的抗病毒疗法的进展。

Progress in RNAi-based antiviral therapeutics.

作者信息

Zhou Jiehua, Rossi John J

机构信息

Division of Molecular and Cellular Biology, Irell and Manella Graduate School of Biological Sciences, Beckman Research Institute of City of Hope, City of Hope, Duarte, CA, USA.

出版信息

Methods Mol Biol. 2011;721:67-75. doi: 10.1007/978-1-61779-037-9_4.

DOI:10.1007/978-1-61779-037-9_4

PMID:21431679

Abstract

RNA interference (RNAi) refers to the conserved sequence-specific degradation of message RNA mediated by small interfering (si)RNA duplexes 21-25 nucleotides in length. Given the ability to specifically silence any gene of interest, siRNAs offers several advantages over conventional drugs as potential therapeutic agents for the treatment of human maladies including cancers, genetic disorders, and infectious diseases. Antiviral RNAi strategies have received much attention and several compounds are currently being tested in clinical trials. In particular, the development of siRNA-based HIV (human immunodeficiency virus) therapeutics has progressed rapidly and many recent studies have shown that the use of RNAi could inhibit HIV-1 replication by targeting a number of viral or cellular genes. Therefore, the present chapter mainly focuses on the recent progress of RNAi-based anti-HIV gene therapeutics, with particular attention to molecular targets and delivery strategies of the siRNAs.

摘要

RNA干扰（RNAi）是指由长度为21 - 25个核苷酸的小干扰（si）RNA双链介导的信使RNA的保守序列特异性降解。鉴于能够特异性沉默任何感兴趣的基因，与传统药物相比，siRNA作为治疗包括癌症、遗传疾病和传染病在内的人类疾病的潜在治疗剂具有多个优势。抗病毒RNAi策略备受关注，目前有几种化合物正在进行临床试验。特别是，基于siRNA的HIV（人类免疫缺陷病毒）治疗药物的开发进展迅速，最近的许多研究表明，使用RNAi可以通过靶向多种病毒或细胞基因来抑制HIV - 1复制。因此，本章主要关注基于RNAi的抗HIV基因治疗的最新进展，特别关注siRNA的分子靶点和递送策略。