肝脏特异性转录调控序列的研究进展  

Progress of Researches on Liver-specific Transcriptional Regulatory Sequence

作　　者：王亚莉[1] 唐红(综述)[1] 雷秉钧(审校)[1] 

机构地区：[1]四川大学华西医院感染性疾病中心生物治疗国家重点实验室,四川大学感染性疾病研究室,成都610041

出　　处：《生物医学工程学杂志》2009年第2期443-447,共5页Journal of Biomedical Engineering

基　　金：国家杰出青年基金资助项目(30325036);973课题资助项目(2006CB504302)

摘　　要：基因治疗是近年迅速发展的一种治疗方式,目前制约基因治疗发展的一个主要问题是其安全性和有效性难以保证,即不能达到治疗基因表达的空间、时序和表达水平的精确定位,特别是治疗基因表达的选择性不高,在非靶组织/细胞表达易产生毒副作用,并降低疗效。通过组织/细胞特异性转录调控元件使外源基因准确、有效地表达于特定组织细胞,从而提高基因治疗的有效性和安全性,已成为基因治疗领域的研究热点。目前研究表明,肝组织中的白蛋白基因和甲胎蛋白基因里有肝脏特异性转录调控序列如启动子、增强子,能启动基因仅在肝组织表达,故它们在转基因动物和基因治疗中得到广泛应用,且其联合应用对提高肝脏的特异性和转录活性有一定作用,在基因治疗研究中也得到了很好的应用,为病毒性肝炎、遗传代谢疾病、肿瘤性疾病等与肝脏密切相关疾病的致病机制和基因治疗的研究奠定了基础。本文就肝脏特异性转录调控序列的研究进展进行综述。Gene therapy is a fast developing therapeutics in recent years. Now, the point at issue that restricts the development of gene therapy is the safety and effectivity of gene expression in space, time series and location. Tissular or cellular specific transcriptional regulatory element can make precise and effective expression of exogenous gene in specified tissue and cell, thus increasing the safety and effectivity of gene expression. This has been a hot spot in gene therapy. Now, researches indicate that hepatic tissue has liver-specific transcriptional regulatory sequence. The regulatory sequences can promote gene expression only in hepatic tissue; they are widely used in transgenic animal and gene therapy. They can serve as a basis for the researches in the pathogenic mechanism and gene therapy of liver-related diseases. New achievements in the studies on liver-specific transcriptional regulatory sequence are reviewed in this article.

关 键 词：基因治疗 肝脏特异性转录调控序列 

分 类 号：R346[医药卫生—基础医学]