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核酶、脱氧核酶和小干扰RNA作为治疗手段。

Ribozymes, DNAzymes and small interfering RNAs as therapeutics.

作者信息

Sioud Mouldy, Iversen Per Ole

机构信息

Institute for Cancer Research, Department of Immunology, Molecular Medicine Group, The Norwegian Radium Hospital, Montebello, 0310 Oslo, Norway.

出版信息

Curr Drug Targets. 2005 Sep;6(6):647-53. doi: 10.2174/1389450054863653.

Abstract

Selective gene silencing by nucleic acid enzymes has provided researchers with a new strategy to block gene expression and drug target validation. Ribozymes, DNAzymes and small interfering RNAs (siRNAs) are being explored as genetic inhibitors of gene expression as well as potential therapeutics against viral infections, inflammatory disorders, haematological diseases and cancers. We review the mode of action of these molecules, with special emphasis on their construction and the possibility to enhance their serum half-lives via specific chemical modifications. Their potential use in cell cultures and in animal models for disease is also highlighted.

摘要

核酸酶介导的选择性基因沉默为研究人员提供了一种阻断基因表达和进行药物靶点验证的新策略。核酶、脱氧核酶和小干扰RNA(siRNA)正在作为基因表达的遗传抑制剂以及针对病毒感染、炎症性疾病、血液系统疾病和癌症的潜在治疗药物进行探索。我们综述了这些分子的作用模式,特别强调了它们的构建以及通过特定化学修饰延长其血清半衰期的可能性。还重点介绍了它们在细胞培养和疾病动物模型中的潜在应用。

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