吗啉反义寡核苷酸在基因功能研究中的应用

The application of phosphorodiamidate morpholino oligomers in the research of gene function

吗啉反义寡核苷酸属于第三代反义寡核苷酸,主要通过阻断mRNA的剪接过程来抑制目的基因的功能。吗啉反义寡核苷酸技术现已广泛应用于发育过程中基因功能的研究;鉴于吗啉反义寡核苷酸能与病毒特异mRNA结合,形成的双链物可有效阻断病毒RNA的转录,从而抑制病毒的复制,所以该技术已应用于医学研究,如治疗病毒感染、癌症、肌营养不良症和早老综合症等疾病。主要阐述了吗啉反义寡核苷酸的结构特点、作用机制、与其它反义技术的比较,以及该技术的应用与展望。

Phosphorodiamidate morpholino oligomers are belong to the third generation of antisense oligonucleotides, and can inhibit tile function of the target gene mainly by blocking mRNA splicing. Morpholino antisense oligonucleotide technology was widely used in the research of gene function during developmental process. The phosphorodiamidate morpholino oligomers and the specific viral mRNA can form the double-stranded material, which effectively block the transcription of viral RNA, and therefore inhibit the viral replica tion. So this technology is also used in the medical research such as treating viral infections, cancer, muscle nutrition dysplasia syndrome and Alzheimer disease. This article focused on the structure and functional mechanism of phosphorodiamidate morpholino oligomers, comparison with other antisense technologies, the applications and prospects of the technology.