基于腺相关病毒载体的人淋巴细胞基因转移。

C A Muro-Cacho, R J Samulski, D Kaplan
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引用次数: 44

摘要

腺相关病毒是一种非致病性、依赖性的细小病毒,整合在人类19号染色体的特定位点。我们利用介导整合的病毒末端反向重复序列,建立了在人淋巴细胞中进行基因转移的载体。一种新霉素耐药基因已被稳定地引入到未转化的人类t细胞克隆中,随后对受感染克隆的功能特性分析显示没有可检测到的改变。野生型病毒的拯救和复制是通过腺病毒的重复感染完成的;然而,该方法没有挽救载体,也没有复制载体,这表明了整合载体的稳定性,并证明了在其构建中纳入了额外的安全性。基于腺相关病毒的载体是淋巴细胞基因治疗中逆转录病毒的替代方法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Gene transfer in human lymphocytes using a vector based on adeno-associated virus.

Adeno-associated virus is a nonpathogenic, dependent parvovirus that integrates at a specific site in human chromosome 19. We have used the inverted terminal repeats of the virus, which mediate integration, to establish a vector for gene transfer in human lymphocytes. A neomycin resistance gene has been stably introduced into nontransformed human T-cell clones and a subsequent analysis of the functional properties of the infected clone revealed no detectable alterations. Rescue and replication of the wild-type virus was accomplished with adenovirus superinfection; however, the vector was not rescued and did not replicate by this procedure, indicating the stability of the integrated vector and demonstrating an additional level of safety incorporated in its construction. An adeno-associated virus-based vector represents an alternative to retroviruses for gene therapy in lymphocytes.

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