Isaka Yoshitaka
Osaka University Graduate School of Medicine, Department of Advanced Technology for Transplantation, Suita 565-0871, Osaka, Japan.
Curr Opin Mol Ther. 2007 Apr;9(2):132-6.
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.
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。这些强效疗法包括含有转基因的质粒、反义寡核苷酸、适体、核酶、脱氧核酶和小干扰RNA。脱氧核酶能够以序列特异性方式切割RNA底物，已被作为靶向致病mRNA的潜在疗法进行研究，可能会产生比现有药物更安全的药物。这篇综述讨论了脱氧核酶的结构设计及其为提高稳定性而进行的修饰。还讨论了它们的治疗应用以及作为生物标志物的用途。
