治疗的意义:设计一种广谱的艾滋病基因疗法。

Current opinion in HIV and AIDS Pub Date : 2024-05-01 Epub Date: 2024-03-01 DOI:10.1097/COH.0000000000000846
Rachel E Berman, Will Dampier, Michael R Nonnemacher, Brian Wigdahl
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引用次数: 0

摘要

综述的目的:治疗人类免疫缺陷病毒 1 型(HIV-1)的主要基因编辑策略包括在腺相关病毒(AAV)载体中递送 SaCas9 和两个引导 RNA(gRNA)。作为一种双组分系统,CRISPR 通过选择 Cas 效应器和 gRNA 原载体设计对靶向遗传位点。近年来,随着CRISPR研究的不断扩展,人们对这些元件在治疗策略中的应用进行了研究,本文将对此进行讨论:迄今为止,II 型 SpCas9 和 SaCas9 一直是基因编辑疗法中的主要 Cas 效应器。此外,广泛的研究拓展了多重 gRNA 的潜力,并将它们有效地靶向高度基因多样化的 HIV-1 病毒。摘要:在了解 CRISPR/Cas 治疗 HIV-1 的各个组成部分时,重要的是要知道目前使用的策略还可以改进。未来的研究领域将包括替代性较小的 Cas 效应体、多重 gRNAs 设计和/或替代性递送模式。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
What's in a cure: designing a broad-spectrum HIV gene therapy.

Purpose of review: The leading gene editing strategy for a human immunodeficiency virus type 1 (HIV-1) cure involves the delivery of SaCas9 and two guide RNAs (gRNAs) in an adeno-associated viral (AAV) vector. As a dual-component system, CRISPR is targeted to a genetic locus through the choice of a Cas effector and gRNA protospacer design pair. As CRISPR research has expanded in recent years, these components have been investigated for utilization in cure strategies, which will be discussed in this article.

Recent findings: Type II SpCas9 and SaCas9 have been the leading Cas effectors across gene editing therapeutics to date. Additionally, extensive research has expanded the potential to multiplex gRNAs and target them effectively to the highly genetically diverse HIV-1 provirus. More recently, the Type V family of Cas12 effectors opens a new opportunity to use a smaller Cas protein for packaging into an AAV vector with multiplexed gRNAs.

Summary: In understanding the individual components of a CRISPR/Cas therapeutic cure for HIV-1, it is important to know that the currently used strategies can be improved upon. Future areas will include alternative smaller Cas effectors, multiplexed gRNAs designs, and/or alternative delivery modalities.

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