Development of hammerhead ribozymes for HIV-1gene therapy: principles and progress

Dr.; Sadhna Joshi

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Development of hammerhead ribozymes for HIV-1gene therapy: principles and progress

机译：用于HIV-1基因治疗的锤头状核酶的开发：原理和进展

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Hammerhead ribozymes are small RNA molecules that can be designed to specifically recognize and cleave a target RNA. A single ribozyme can theoretically act in a catalytic manner thus cleaving more than one molecule of its target RNA. Because of their sequence specificity, ribozymes are being developed as therapeutic agents to eliminate unwanted cellular and viral RNAs. Ribozymes are being used to inhibit human immunodeficiency virus type-1 (HIV-1) replication. Promising results have been obtained by several groups using ribozymes targeted against various sites within the HIV-1 genome. This article reviews some of the factors relevant to the design of hammerhead ribozymes with a particular focus on their application in HIV-1 gene therapy.

机译：锤头状核酶是小的RNA分子，可以设计为特异性识别和切割靶RNA。理论上，单个核酶可以以催化方式发挥作用，从而裂解一个以上的靶RNA分子。由于它们的序列特异性，正在开发核酶作为治疗剂以消除不需要的细胞和病毒RNA。核酶正用于抑制人类免疫缺陷病毒1型（HIV-1）复制。几组使用针对HIV-1基因组内各种位点的核酶已获得了可喜的结果。本文回顾了与锤头状核酶设计有关的一些因素，尤其着重于它们在HIV-1基因治疗中的应用。

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《Gene Therapy and Molecular Biology》 |1999年第2期|共10页
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Dr.; Sadhna Joshi;
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Development of hammerhead ribozymes for HIV-1gene therapy: principles and progress

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