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重组腺病毒伴随病毒(Adeno-associated virus,AAV)载体是能建立稳定表达的转导型表达载体,可用作基因治疗的外源基因荷载工具.与逆转录病毒相比,它具有高安全性、无致病性和能感染有丝分裂后细胞等的特点.目前重组AAV的制备,需要两种不同的重组AAV质粒,一种是含位于AAV基因组两端的为病毒复制和包装所必需的反向末端重复(Inverted ter-minal repeats,ITR)和外源基因的重组表达质粒载体;另一种质粒作为助手质粒,克隆了除两端ITR外的AAV全基因组DNA,它反式提供AAV复制和包装所需的病毒蛋白,当这两种质粒共转染宿主细胞时,如存在辅助病毒(腺病毒或单纯疱疹病毒)的感染,就能包装出含外源基因的重组AAV假病毒颗粒.
The Adeno-associated virus (AAV) vector is a transformant expression vector which can be used as a gene expression vector for gene therapy, which has high safety compared with retrovirus , Non-pathogenic and capable of infecting mitotic cells, etc. Currently, two different recombinant AAV plasmids are required for the production of recombinant AAV, one containing the reverse termini necessary for viral replication and packaging at both ends of the AAV genome Inverted ter-minal repeats (ITRs) and exogenous gene recombination plasmid vectors were constructed. The other plasmid, a helper plasmid, cloned the AAV genome DNA except for the two-terminal ITR and provided the AAV replication and packaging site When these two plasmids are co-transfected into a host cell, recombinant AAV pseudovirions containing exogenous genes can be packaged in the presence of a helper virus (adenovirus or herpes simplex virus) infection.