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基于 RNA 的基因治疗用于治疗和预防 HIV:从实验室到临床。

RNA-based gene therapy for the treatment and prevention of HIV: from bench to bedside.

机构信息

Department of Internal Medicine, Section of Infectious Diseases, Yale School of Medicine, New Haven, Connecticut 06511, USA.

出版信息

Yale J Biol Med. 2011 Sep;84(3):301-9.

Abstract

Gene therapy is considered a feasible approach for the treatment and prevention of HIV/AIDS. Targeting both viral genes and host dependency factors can interfere with the viral lifecycle and prevent viral replication. A number of approaches have been taken to target these genes, including ribozymes, aptamers, and RNAi based therapies. A number of these therapies are now beginning to make their way into clinical trials and providing proof of principle that gene therapy is a safe and realistic option for treating HIV. Here, we focus on those therapies that have progressed along the pipeline to preclinical and clinical testing.

摘要

基因治疗被认为是治疗和预防 HIV/AIDS 的一种可行方法。针对病毒基因和宿主依赖因素都可以干扰病毒生命周期并阻止病毒复制。已经采取了多种方法来针对这些基因,包括核酶、适体和基于 RNAi 的疗法。其中一些疗法现在开始进入临床试验,并提供了基因治疗是治疗 HIV 的一种安全且现实的选择的原理证明。在这里,我们专注于那些沿着研发管道进展到临床前和临床测试的疗法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f056/3178861/95636bf45f07/yjbm_84_3_301_g01.jpg

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