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腺相关病毒作为人类基因治疗的病毒载体。重组病毒的产生。

AAV as a viral vector for human gene therapy. Generation of recombinant virus.

作者信息

Rolling F, Samulski R J

机构信息

Gene Therapy Center, University of North Carolina at Chapel Hill 27599-7352, USA.

出版信息

Mol Biotechnol. 1995 Feb;3(1):9-15. doi: 10.1007/BF02821330.

Abstract

Investigation of the adeno-associated virus (AAV) life cycle has enabled the establishment of methodology and identification of critical cis-acting sequences required for recombinant AAV production. Vectors derived from the defective human parvovirus (AAV) have been used for successful gene transfer and expression in many diverse mammalian cell types, such as erythroid, airway epithelium, and neuronal cells. One of the crucial steps in the continued case of AAV as a vector is the development of packaging systems that will allow efficient encapsidation of foreign genes into AAV virions. For this reason, the focus of this article will be generation of recombinant AAV vectors.

摘要

对腺相关病毒(AAV)生命周期的研究使得用于重组AAV生产的方法得以建立,并确定了关键的顺式作用序列。源自缺陷型人细小病毒(AAV)的载体已成功用于多种不同的哺乳动物细胞类型(如红细胞、气道上皮细胞和神经元细胞)中的基因转移和表达。在AAV作为载体的持续应用中,关键步骤之一是开发包装系统,该系统能够将外源基因高效包装到AAV病毒粒子中。因此,本文将重点关注重组AAV载体的生成。

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