Adenovirus-Adeno-Associated Virus Hybrid for Large-Scale Recombinant Adeno-Associated Virus Production

被引:31
|
作者
Zhang, Hongwei [1 ]
Xie, Jun [1 ]
Xie, Qing [1 ,2 ]
Wilson, James M. [3 ]
Gao, Guangping [1 ]
机构
[1] Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA 01605 USA
[2] Peking Univ, Hlth Sci Ctr, Dept Microbiol, Beijing 100191, Peoples R China
[3] Univ Penn, Sch Med, Gene Therapy Program, Philadelphia, PA 19104 USA
关键词
HUMAN-PAPILLOMAVIRUS TYPE-16; CELL-LINES; GENE AMPLIFICATION; IN-VITRO; EFFICIENT TRANSDUCTION; VECTORS; AAV; REP; REPLICATION; PURIFICATION;
D O I
10.1089/hum.2009.125
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) holds promise for applications in gene therapy. Advances in clinical studies of rAAV-based gene therapeutics have generated an encouraging momentum in the field of gene therapy; however, one of the major obstacles to the eventual clinical success of rAAV-mediated gene therapy is the need for large-scale production of clinical-grade vectors. The transfection-based rAAV production method is well suited for preclinical studies in small animal models, but it is difficult to support large-scale clinical studies with this method. In the past decade, several scalable rAAV production methods have emerged from extensive efforts to develop large-scale manufacturing processes. Among those, the recombinant adenovirus-AAV infection method has some unique features in vector quality and yield. This minireview provides an overview of this scaleable rAAV production platform, describing its basic components and biological mechanisms and process.
引用
收藏
页码:922 / 929
页数:8
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