AAV 介导的基因治疗用于研究和治疗目的。
AAV-Mediated Gene Therapy for Research and Therapeutic Purposes.
机构信息
Gene Therapy Center, University of North Carolina, Chapel Hill, North Carolina 27599; email:
Powell Gene Therapy Center, College of Medicine, University of Florida, Gainesville, Florida 32610; email:
出版信息
Annu Rev Virol. 2014 Nov;1(1):427-51. doi: 10.1146/annurev-virology-031413-085355.
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.
腺相关病毒（AAV）是一种小型、无包膜病毒，30 年前被改造用于作为基因转移载体。它能够在体内转导广泛的物种和组织，没有毒性的证据，并且产生相对温和的先天和适应性免疫反应。我们回顾了 AAV 的基础生物学、AAV 载体技术的发展历史，以及 AAV 在一些成功的临床和研究应用。
Zotero 插件