RNA-based therapeutics: current progress and future prospects.
RNA-based therapeutics: current progress and future prospects.
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DOI：
10.1016/j.chembiol.2011.12.008
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发表时间：
2012-01-27
影响因子：
--
通讯作者：
Rossi JJ
中科院分区：
文献类型：
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作者：
Burnett JC;Rossi JJ
生物药物的最新进展拓宽了各种人类疾病的治疗靶点的范围。这适用于目前正在进行临床研究的数十种基于RNA的疗法，这些疗法适用于从遗传疾病到艾滋病毒感染到各种癌症的各种疾病。这些新兴药物，包括治疗性核酶、适配子和小干扰RNA(SiRNAs)，展示了RNA前所未有的多功能性。然而，RNA在本质上是不稳定的，具有潜在的免疫原性，通常需要一种运输工具才能有效地运送到靶细胞。这些问题阻碍了一些基于RNA的药物的临床进展，并导致临床测试结果喜忧参半。然而，最近临床试验的有希望的结果表明，通过改进合成递送载体和对RNA疗法进行化学修饰，这些障碍可能被克服。本文就siRNA、RNA适配子和核酶治疗的临床效果及未来的发展前景进行综述。
Recent advances of biological drugs have broadened the scope of therapeutic targets for a variety of human diseases. This holds true for dozens of RNA-based therapeutics currently under clinical investigation for diseases ranging from genetic disorders to HIV infection to various cancers. These emerging drugs, which include therapeutic ribozymes, aptamers, and small interfering RNAs (siRNAs), demonstrate the unprecedented versatility of RNA. However, RNA is inherently unstable, potentially immunogenic, and typically requires a delivery vehicle for efficient transport to the targeted cells. These issues have hindered the clinical progress of some RNA-based drugs and have contributed to mixed results in clinical testing. Nevertheless, promising results from recent clinical trials suggest that these barriers may be overcome with improved synthetic delivery carriers and chemical modifications of the RNA therapeutics. This review focuses on the clinical results of siRNA, RNA aptamer, and ribozyme therapeutics and the prospects for future successes.
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影响因子：
64.8
作者：
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通讯作者：
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