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出 处:《国际遗传学杂志》2007年第5期369-373,共5页International Journal of Genetics
基 金:国家自然科学基金资助项目(30571068);973项目的资助(2004CB518802)
摘 要:添加、修复或替换基因从而达到直接排除病因是基因治疗的目的,也是用于治疗遗传性和获得性疾病的治疗方法。它包括目的基因、载体和靶细胞三个方面,其中载体在整个传染过程中起着关键的作用。非病毒载体是该研究领域的热点之一,虽然传染效率不如病毒载体,但其无毒、无免疫反应、性质可调且制备方便。该文主要就非病毒载体的转染机制及优化策略作一综述。The ultimate goal of gene therapy is to eure both inberited and acquired disorders in a straight forward manner by removing their cause, that is, by adding, correcting, or replacing gene. Gene therapy is promising for curing various inherted of acquired diseases. It including three aspects:therapeutic gene, vector and target cell. Among which the vector plays an important role in the course of transfection. Nonvirus vector is the hot-spot of reseach in this field. Compared with viral vectors, the case of manipulation, no toxicity, no immunoreaction,and high flexibility regarding the size of the transgene delivered make nonviral vectors more and more attractive.
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