PDF(Pubmed)
Abstract：
Based on the development of nucleic acid therapeutic drugs, DNAzymes obtained through in vitro selection technology in 1994 are gradually being sought. DNAzymes are single-stranded DNA molecules with catalytic function, which specifically cleave RNA under the action of metal ions. Various in vivo and in vitro models have recently demonstrated that DNAzymes can target related genes in cancer, cardiovascular disease, bacterial and viral infection, and central nervous system disease. Compared with other nucleic acid therapy drugs, DNAzymes have gained more attention due to their excellent cutting efficiency, high stability, and low cost. Here, We first briefly reviewed the development and characteristics of DNAzymes, then discussed disease-targeting inhibition model of DNAzymes, hoping to provide new insights and ways for disease treatment. Finally, DNAzymes were still subject to some restrictions in practical applications, including low cell uptake efficiency, nuclease degradation and interference from other biological matrices. We discussed the latest delivery strategy of DNAzymes, among which lipid nanoparticles have recently received widespread attention due to the successful delivery of the COVID-19 mRNA vaccine, which provides the possibility for the subsequent clinical application of DNAzymes. In addition, the future development of DNAzymes was prospected.
摘要：
基于核酸治疗药物的发展，在1994年通过体外选择技术获得的DNAzyme正在逐渐被寻找。DNA酶是具有催化功能的单链DNA分子,在金属离子的作用下特异性切割RNA。各种体内和体外模型最近证明DNA酶可以靶向癌症中的相关基因。心血管疾病,细菌和病毒感染，和中枢神经系统疾病。与其他核酸治疗药物相比，DNAzyme由于其优异的切割效率而获得了更多的关注，高稳定性,和低成本。这里,我们首先简要回顾了DNAzyme的发展和特点，然后讨论了DNAzymes的疾病靶向抑制模型，希望为疾病治疗提供新的见解和方法。最后,DNA酶在实际应用中仍然受到一些限制，包括低细胞摄取效率，核酸酶降解和其他生物基质的干扰。我们讨论了DNAzymes的最新交付策略，其中脂质纳米粒最近由于COVID-19mRNA疫苗的成功递送而受到广泛关注，这为DNAzymes的后续临床应用提供了可能性。此外,对DNAzyme的未来发展进行了展望。
