DNAzymes as potential therapeutic molecules.
DNAzymes as potential therapeutic molecules.
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发表时间：
2007-04
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通讯作者：
Y. Isaka
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作者：
Y. Isaka
许多基于 DNA 的生物制药能够通过诱导和/或抑制基因来控制疾病进展。这些有效的治疗方法包括含有转基因的质粒、反义寡核苷酸、适体、核酶、脱氧核糖核酸酶和短干扰RNA。 DNAzymes 以序列特异性方式切割 RNA 底物，已被研究作为针对致病性 mRNA 的潜在疗法，并且可能产生比目前可用的药物更安全的药物。这篇综述讨论了 DNAzyme 的结构设计及其稳定性修饰。还讨论了它们的治疗应用和作为生物标志物的用途。
Numerous DNA-based biopharmaceuticals are able to control disease progression by induction and/or inhibition of genes. These potent therapeutics include plasmids containing transgenes, antisense oligonucleotides, aptamers, ribozymes, DNAzymes and short interfering RNAs. DNAzymes, which cleave RNA substrates in a sequence-specific manner, have been studied as potential therapeutics to target pathogenic mRNAs, and may yield drugs that are safer than those currently available. This reviewo discusses the structural design of DNAzymes and their modification for stability. Their therapeutic application and use as biomarkers is also discussed.