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AAV-Mediated Gene Therapy for Research and Therapeutic Purposes.
Annual Review of Virology ( IF 8.1 ) Pub Date : 2014-11-03 , DOI: 10.1146/annurev-virology-031413-085355
R Jude Samulski 1 , Nicholas Muzyczka 2
Affiliation  

Adeno-associated virus (AAV) is a small, nonenveloped virus that was adapted 30 years ago for use as a gene transfer vehicle. It is capable of transducing a wide range of species and tissues in vivo with no evidence of toxicity, and it generates relatively mild innate and adaptive immune responses. We review the basic biology of AAV, the history of progress in AAV vector technology, and some of the clinical and research applications where AAV has shown success.

中文翻译:

用于研究和治疗目的的AAV介导的基因治疗。

腺伴随病毒(AAV)是一种小型的无包膜病毒,在30年前被改编为基因转移载体。它能够在体内转导各种各样的物种和组织,而没有毒性的证据,并且它产生相对温和的先天性和适应性免疫反应。我们回顾了AAV的基本生物学,AAV载体技术的发展历史以及AAV取得成功的一些临床和研究应用。
更新日期:2014-10-01
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