Production of recombinant adeno-associated virus vectors

被引：37

作者：

Zolotukhin, S ^{[1
]}

机构：

[1] Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, Alachua, FL 32615 USA

来源：

HUMAN GENE THERAPY | 2005年 / 16卷 / 05期

关键词：

D O I：

10.1089/hum.2005.16.551

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

Recombinant adeno-associated virus (rAAV) is a prototypical gene therapy vector characterized by excellent safety profiles, wide host range, and the ability to transduce differentiated cells. Numerous rAAV-based vectors providing efficient and sustained expression of transgenes in target tissues have been developed for preclinical studies. Interest in rAAV has been driven by advances in production methods originally developed for rAAV serotype 2 vectors and expanded to include alternative serotypes. The transition to clinical trials is dependent on the development of scalable production methods of Good Manufacturing Practice-grade vectors described in this review.

引用

页码：551 / 557

页数：7

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