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AAV载体靶向修饰策略在基因治疗中的研究进展

     

摘要

Adeno?associated virus( AAV) belongs to the family of Parvoviridae. It is a small virus which can infect humans and some other primate species with extensive host cell and low?immunogenicity. Besides, modified AAV can target the specific tissues and cells effectively. As a kind of gene therapy vector,Adeno?associated virus have been widely known of its biological characteristics. By modification of the adeno?associated virus serotype and structure of capsid protein, we can extend the application range of AAV vectors. This article introduces the targeting mechanism and results of some related researches, the genetic engineering modification of AAV capsid proteins, the transcription regulation modification of target gene expression, and gene modification and the covalent coupling of AAV capsid proteins.%腺相关病毒(adeno?associated virus,AAV)是微小病毒科的一种,它能以其广泛的宿主范围及低的免疫原性对人及灵长类进行感染,而且经过改造后的AAV能够更有效的转导特异组织及细胞.腺相关病毒作为一种基因治疗载体其生物学特性已被深入了解,通过改造腺相关病毒的血清型和蛋白衣壳结构能够扩宽其应用范围.本文对AAV病毒载体的衣壳蛋白基因工程修饰、转录水平调控修饰、基因改造和衣壳蛋白共价偶联修饰的靶向机理以及相关的一些研究成果进行介绍.

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