Gene Therapy Strategies to Block HIV-1 Replication by RNA Interference
Gene Therapy Strategies to Block HIV-1 Replication by RNA Interference
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DOI：
10.1007/978-1-4939-2432-5_4
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发表时间：
2015-01-01
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影响因子：
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通讯作者：
Berkhout, Ben
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文献类型：
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作者：
Herrera-Carrillo, Elena;Berkhout, Ben
RNA干扰（RNAi）的细胞机制在许多生物体中起着抗病毒作用，并可用于开发针对病毒病原体的治疗策略。持续性感染，如由人类免疫缺陷病毒1型（HIV-1）引起的感染，可能需要持久的基因治疗方法。抑制性RNA分子在T细胞中的持续表达是有效阻断HIV-1复制所必需的。我们在这里讨论了几个问题，从RNAi抑制剂和载体系统的选择，在HIV-1 RNA基因组中找到最佳靶点，或者通过靶向编码重要病毒辅因子的宿主mRNA，建立适当的临床前测试系统。最后，我们简要讨论了其他病毒性病原体，导致人类慢性感染的相关性。
The cellular mechanism of RNA interference (RNAi) plays an antiviral role in many organisms and can be used for the development of therapeutic strategies against viral pathogens. Persistent infections like the one caused by the human immunodeficiency virus type 1 (HIV-1) likely require a durable gene therapy approach. The continuous expression of the inhibitory RNA molecules in T cells is needed to effectively block HIV-1 replication. We discuss here several issues, ranging from the choice of RNAi inhibitor and vector system, finding the best target in the HIV-1 RNA genome, alternatively by targeting host mRNAs that encode important viral cofactors, to the setup of appropriate preclinical test systems. Finally, we briefly discuss the relevance of this topic for other viral pathogens that cause a chronic infection in humans.