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CRISPR therapy towards an HIV cure.
Briefings in Functional Genomics ( IF 2.5 ) Pub Date : 2020-05-20 , DOI: 10.1093/bfgp/elz021
Elena Herrera-Carrillo 1 , Zongliang Gao 1 , Ben Berkhout 1, 2
Affiliation  

Tools based on RNA interference (RNAi) and the recently developed clustered regularly short palindromic repeats (CRISPR) system enable the selective modification of gene expression, which also makes them attractive therapeutic reagents for combating HIV infection and other infectious diseases. Several parallels can be drawn between the RNAi and CRISPR-Cas9 platforms. An ideal RNAi or CRISPR-Cas9 therapeutic strategy for treating infectious or genetic diseases should exhibit potency, high specificity and safety. However, therapeutic applications of RNAi and CRISPR-Cas9 have been challenged by several major limitations, some of which can be overcome by optimal design of the therapy or the design of improved reagents. In this review, we will discuss some advantages and limitations of anti-HIV strategies based on RNAi and CRISPR-Cas9 with a focus on the efficiency, specificity, off-target effects and delivery methods.

中文翻译:

针对HIV治愈的CRISPR疗法。

基于RNA干扰(RNAi)的工具和最近开发的聚类的规则的短回文重复序列(CRISPR)系统可以选择性修饰基因表达,这也使它们成为抗击HIV感染和其他传染病的有吸引力的治疗剂。RNAi和CRISPR-Cas9平台之间可以得出一些相似之处。治疗传染性或遗传性疾病的理想RNAi或CRISPR-Cas9治疗策略应表现出效力,高特异性和安全性。但是,RNAi和CRISPR-Cas9的治疗应用受到了几个主要限制的挑战,其中一些可以通过优化治疗方法或设计改良试剂来克服。在这篇评论中
更新日期:2019-11-08
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