Adeno-associated Virus as a Mammalian DNA Vector.
Adeno-associated Virus as a Mammalian DNA Vector.
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DOI：
10.1128/microbiolspec.mdna3-0052-2014
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发表时间：
2015-08
影响因子：
3.7
通讯作者：
Samulski RJ
中科院分区：
文献类型：
--
作者：
Salganik M;Hirsch ML;Samulski RJ
腺相关病毒（adeno-associated virus， AAV）自意外发现以来的近50年里，已经成为一种高度通用的载体系统，用于研究和临床应用。广泛的自然血清型，以及越来越多的衣壳变异，结合起来产生了一系列具有不同组织趋向性、免疫原性和转导效率的载体。AAV的故事是病毒系统持续进步和惊人发现的故事之一，乍一看，它看似简单。这种明显的简单性使得AAV进入临床，尽管存在一些挑战，但它为患者提供了希望，并为医生提供了一个有前途的新工具。尽管在研究AAV的基础生物学和优化其临床应用方面仍有大量工作要做，但AAV载体是目前在哺乳动物细胞中最安全、最有效的基因转移平台。
In the nearly five decades since its accidental discovery, adeno-associated virus (AAV) has emerged as a highly versatile vector system for both research and clinical applications. A broad range of natural serotypes, as well as an increasing number of capsid variants, has combined to produce a repertoire of vectors with different tissue tropisms, immunogenic profiles and transduction efficiencies. The story of AAV is one of continued progress and surprising discoveries in a viral system that, at first glance, is deceptively simple. This apparent simplicity has enabled the advancement of AAV into the clinic, where despite some challenges it has provided hope for patients and a promising new tool for physicians. Although a great deal of work remains to be done, both in studying the basic biology of AAV and in optimizing its clinical application, AAV vectors are currently the safest and most efficient platform for gene transfer in mammalian cells.