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首页> 外文期刊>Chinese science bulletin >A novel package system based on an EBV replicon vector for producing high titer recombinant adeno-associated virus vector
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A novel package system based on an EBV replicon vector for producing high titer recombinant adeno-associated virus vector

机译：一种基于EBV复制子载体的新型包装系统，用于生产高滴度重组腺相关病毒载体

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ADENO-ASSOCIATED virus (AAV) was a human parvovirus considered as a gene delivery vehicle for human gene therapy. Recombinant AAV vector (rAAV) could mediate the foreign DNA integration into chromosome and establish a stable expression in the infectedcells. As a viral transduction vector, rAAV was superior to the traditional retrovirus vector for its high safety, no pathogenicity, and capacity of infecting postmitosis cells. The current strategy for producing rAAV involves two recombinant plasmids.One is the expression vector harboring a foreign gene expression cassette flanking with the AAV-inverted terminal repeats (ITR), which are necessary for viral replication and packaging; the other is the complementing plasmid (helper plasmid), which is defective for packaging but supplies the wild type AAV gene products. When host cells were trnasfected with these two plasmids and infected with Adenovirus of Herpes virus to provide the helper function, rAAV particles could be produced.
机译：ADENO-ASSOCIATED病毒（AAV）是一种人类细小病毒，被认为是人类基因治疗的基因传递载体。重组AAV载体（rAAV）可以介导外源DNA整合入染色体并在感染细胞中建立稳定的表达。作为一种病毒转导载体，rAAV具有较高的安全性，无致病性和感染有丝分裂后细胞的能力，优于传统的逆转录病毒载体。目前生产rAAV的策略涉及两个重组质粒。一个是带有外源基因表达盒的表达载体，该表达盒的侧面是AAV反向末端重复序列（ITR），这对于病毒复制和包装是必需的。另一个是互补质粒（辅助质粒），该质粒包装有缺陷，但可以提供野生型AAV基因产物。当用这两个质粒转染宿主细胞并用疱疹病毒腺病毒感染以提供辅助功能时，可以产生rAAV颗粒。

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