Adeno-associated virus vectors: versatile tools for in vivo gene transfer.
Adeno-associated virus vectors: versatile tools for in vivo gene transfer.
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DOI：
10.1159/000125584
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发表时间：
2008
影响因子：
--
通讯作者：
L. Zentilin;M. Giacca
中科院分区：
文献类型：
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作者：
L. Zentilin;M. Giacca
在过去的几年里，基于腺相关病毒的病毒载体由于几个有利的特性而越来越受欢迎，包括有丝分裂后组织在体内的高效转导以及在没有炎症或免疫反应的情况下转基因表达的长期持久性。最近，完成的试验基本上证实了这些媒介的临床适用性，同时表明需要进一步发展媒介设计和生产规模，以扩大人类应用。本文综述了这些载体的分子生物学及其临床应用，特别是它们在肾脏基因转移中的应用。
Over the last few years, viral vectors based on the adeno-associated virus have gained increasing popularity due to several favorable characteristics, including the high efficiency of transduction of postmitotic tissues in vivo and the long-term persistence of transgene expression in the absence of inflammation or immune response. Recently, completed trials have substantially confirmed the clinical applicability of these vectors, while indicating that further developments in vector design and production scale are necessary to broaden human application. This review summarizes our current knowledge on the molecular biology of these vectors and their clinical utilization, in particular concerning their application in renal gene transfer.