摘要
在人类进入后基因组时代的今天 ,基因治疗将会成为与各种遗传和非遗传性疾病做斗争的一种有力武器。有效的基因治疗取决于对疾病发生分子机制的正确认识。随着对动脉粥样硬化发病机理认识的深入 ,一些分子靶点被逐步确认。其中系统靶点以降低低密度脂蛋白 (LDL)和影响高密度脂蛋白 (HDL)代谢为主 ,局部靶点则主要为抑制动脉壁平滑肌细胞增殖、局部抗血栓、抗氧化和促进血管内皮修复。针对这一系列靶点 ,有的基因需要高表达 ,有的基因则需要抑制 ,才能起到治疗作用。但是目前针对动脉粥样硬化的基因治疗还主要在实验室研究阶段 ,向临床应用的过渡还有赖于基因载体系统的研发和最适动物模型的制备。本文对上述问题进行了较为详细的讨论。
Effective gene therapy for dyslipidemia and atherosclerosis primarily depends on an appropriate selection of therapeutic targets. Different forms of dyslipidemia are the major causes for atherosclerosis, which can be classified as increased LDL levels and altered HDL metabolism. These disorders could be treated by gene delivery to systemic targets. Local targets are mainly composed of pathological processes at the sites of atherosclerotic lesion, such as proliferation of smooth muscle cells, endothelial dysfunction and localized thrombosis. The expression of the genes at these sites should be either up regulated via gene delivery or inhibited by anti sense technology. The ideal delivery vector system has yet to be developed and approprate animal models are required before gene therapy for dyslipidemia and atherosclerosis can be successfully applied in patients.
出处
《基础医学与临床》
CSCD
北大核心
2002年第6期498-503,共6页
Basic and Clinical Medicine
