剪接变异体的肿瘤靶向性研究进展  

Research progress on tumor targeting of splice variants

作　　者：李燕妮[1,2] 姚晖[1,2] 

机构地区：[1]南开大学口腔医院 [2]天津市口腔医院中心实验室,天津市300041

出　　处：《中国肿瘤临床》2014年第3期215-217,共3页Chinese Journal of Clinical Oncology

摘　　要：剪接变异体是mRNA前体选择性剪接过程中形成的不同功能或活性的转录本。随着越来越多的肿瘤特异性剪接变异体的发现,剪接变异体在肿瘤发生发展过程中所发挥的作用也逐渐受到学者的关注。由于肿瘤特异性剪接变异体的表达模式和活性都极具特征性,提示针对靶向肿瘤细胞的剪接体的基因治疗将更具特异性,并对正常组织产生较小的影响。临床试验应用反义寡核苷酸下调了影响肿瘤细胞存活率的mRNA并改变其剪接模式,为肿瘤基因治疗提供了新的思路。随着新的肿瘤特异性剪接变异体的识别以及治疗策略的不断改进,剪接变异体及剪接模式的改变将成为研究肿瘤安全、有效治疗方法的一个重要领域。Splice variants are transcripts that exhibit various fianctions or activities and are obtained from alternative splicing of precursor mRNA. An increasing number of tumor-specific splice variants are being discovered and their roles in cancer development and progression are being investigated. In this study, the expression patterns and activities of tumor-specific splice variants were charac- terized. The results suggest that a variant-specific gene therapy that targets cancer cells would be more specific and would have minimal effect on healthy tissues. Using antisense oligonucleotides, mRNA that contributed to cancer cell survival and altered splicing patterns was downregulated in a clinical trial. The results provide a new appreciation for the gene therapy of cancer. Identification of novel tu- mor-specific splice variants and constant improvement of therapeutic strategies by modifying splicing pattems can lead to the develop- ment of more effective and safer cancer therapies.

关 键 词：剪接变异体 选择性剪接 肿瘤特异性 靶向性 

分 类 号：R730.2[医药卫生—肿瘤]