利用腺相关病毒的基因组工程：基础与临床研究应用
Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications.
作者信息
Gaj Thomas, Epstein Benjamin E, Schaffer David V
机构信息
Department of Chemical and Biomolecular Engineering, University of California, Berkeley, California, USA.
Department of Bioengineering, University of California, Berkeley, California, USA.
出版信息
Mol Ther. 2016 Mar;24(3):458-64. doi: 10.1038/mt.2015.151. Epub 2015 Sep 16.
Abstract
In addition to their broad potential for therapeutic gene delivery, adeno-associated virus (AAV) vectors possess the innate ability to stimulate homologous recombination in mammalian cells at high efficiencies. This process--referred to as AAV-mediated gene targeting--has enabled the introduction of a diverse array of genomic modifications both in vitro and in vivo. With the recent emergence of targeted nucleases, AAV-mediated genome engineering is poised for clinical translation. Here, we review key properties of AAV vectors that underscore its unique utility in genome editing. We highlight the broad range of genome engineering applications facilitated by this technology and discuss the strong potential for unifying AAV with targeted nucleases for next-generation gene therapy.
摘要
除了在治疗性基因递送方面具有广泛潜力外，腺相关病毒（AAV）载体还具有在哺乳动物细胞中高效刺激同源重组的固有能力。这一过程——称为AAV介导的基因靶向——已使得在体外和体内引入了各种各样的基因组修饰。随着靶向核酸酶的近期出现，AAV介导的基因组工程有望实现临床转化。在这里，我们回顾了AAV载体的关键特性，这些特性突出了其在基因组编辑中的独特用途。我们强调了这项技术所推动的广泛基因组工程应用，并讨论了将AAV与靶向核酸酶结合用于下一代基因治疗的强大潜力。
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