基因治疗新策略——酶性DNA的设计和应用  

New strategy in the gene therapy:design and application of catalytic DNA

作　　者：于乐成[1] 顾长海[1] 王升启[2] 毛青[1] 

机构地区：[1]第三军医大学西南医院全军感染病研究所,重庆400038 [2]军事医学科学院放射医学研究所,北京100850

出　　处：《中国药物化学杂志》2002年第4期236-242,共7页Chinese Journal of Medicinal Chemistry

摘　　要：本文综述了能够剪切RNA分子的酶性DNA的种类、结构、作用机制、动力学特征及其在用作新型基因治疗药物方面的优势、设计要点。Catalytic DNAs,also named deoxyribozymes,DNAzymes or DNA enzymes,were synthetic oligodeoxynuckeotides able to specifically bind and cleave different target RNAs.10 23,8 17 and HD2 deoxyribozymes were the main catalytic DNAs that have been found.It was reported that those catalytic DNAs,especially 10 23 deoxyribozyme,could be used to cleave and suppress various mRNAs of pathogenic microbes,malignant tumors and some genetic diseases.Their structures,mechanisms of cleaving RNA,dynamic characteristics,current application were summarized;and the design principle,advantages of using as new gene therapeutic tools,possible application in future were discussed.

关 键 词：基因治疗 酶性DNA 设计 应用 脱氧核酶 药物设计 

分 类 号：R346[医药卫生—基础医学]