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Human Gene Therapy
Isolation of Highly Infectious and Pure Adeno-Associated Virus Type 2 Vectors with a Single-Step Gravity-Flow Column
To cite this article:
Alberto Auricchio, Markus Hildinger, Erin O'Connor, Guang-Ping Gao, James M. Wilson.
Human Gene Therapy.
January 2001,
12(1): 71-76.
doi:10.1089/104303401450988.
Alberto Auricchio Wistar Institute, Philadelphia, PA 19104. Markus Hildinger Wistar Institute, Philadelphia, PA 19104. Erin O'Connor Wistar Institute, Philadelphia, PA 19104. Guang-Ping Gao Wistar Institute, Philadelphia, PA 19104. James M. Wilson Wistar Institute, Philadelphia, PA 19104. One of the most promising gene transfer vectors in human clinical trials is AAV2. The quality of the vector preparations is a key element in obtaining reliable and reproducible data in preclinical studies. However, established protocols either result in impure, low infectious virus (CsCl2 gradient centrifugation) or demand a high level of manual and technical skills (CsCl2 gradient centrifugation, iodixanol/heparin or HPLC purification). In this study, we present an easy-to-do single-step column purification (SSCP) of AAV2 by gravity flow based on its affinity to heparin, without ultracentrifugation. Various vector preparations generated by our method reproducibly showed high titers, infectivity, and purity. In vivo, our single-step column-purified AAV2 vectors mediate significantly higher transduction efficiency compared with conventional protocols. Investigators still unsatisfied with previously published techniques or new to the field of AAV production may find in our method an interesting alternative.  This paper was cited by:The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice Jiangao Zhu, Xiaopei Huang, Yiping Yang Journal of Clinical Investigation. Aug 2009 CrossRef Enhanced transgene expression in the mouse skeletal muscle infected by the adeno-associated viral vector with the human elongation factor 1α promoter and a human chromatin insulator Mayuyo Mori-Uchino, Takamasa Takeuchi, Isao Murakami, Tetsu Yano, Toshiharu Yasugi, Yuji Taketani, Keiichi Nakagawa, Tadahito Kanda The Journal of Gene Medicine. Aug 2009, Vol. 11, No. 7: 598-604 CrossRef A Simplified Baculovirus-AAV Expression Vector System Coupled With One-step Affinity Purification Yields High-titer rAAV Stocks From Insect Cells Richard H Smith, Justin R Levy, Robert M Kotin Molecular Therapy. Jul 2009 CrossRef Local Knockdown of ERK2 in the Adult Mouse Brain Via Adeno-Associated Virus-Mediated RNA Interference Barbara Di Benedetto, Benedikt Wefers, Wolfgang Wurst, Ralf Kühn Molecular Biotechnology. Apr 2009, Vol. 41, No. 3: 263-269 CrossRef Tailoring the AAV vector capsid for gene therapy L H Vandenberghe, J M Wilson, G Gao Gene Therapy. Apr 2009, Vol. 16, No. 3: 311-319 CrossRef Expression of transgenes in midbrain dopamine neurons using the tyrosine hydroxylase promoter M S Oh, S J Hong, Y Huh, K-S Kim Gene Therapy. Apr 2009, Vol. 16, No. 3: 437-440 CrossRef Targeted Overexpression of Human α-Synuclein Triggers Microglial Activation and an Adaptive Immune Response in a Mouse Model of Parkinson Disease Shaji Theodore, Shuwen Cao, Pamela J. McLean, David G. Standaert Journal of Neuropathology and Experimental Neurology. Jan 2009, Vol. 67, No. 12: 1149-1158 CrossRef The four-and-a-half-LIM protein 2 (FHL2) is overexpressed in gliomas and associated with oncogenic activities Ming Li, Jide Wang, Samuel S. M. Ng, Chu-Yan Chan, Amy C. Chen, Hong Ping Xia, David T. Yew, Benjamin C. Y. Wong, Zhu Chen, Hsiang-Fu Kung, Marie Chia-Mi Lin Glia. Oct 2008, Vol. 56, No. 12: 1328-1338 CrossRef Strategies for improving the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo G R Jayandharan, L Zhong, B Li, B Kachniarz, A Srivastava Gene Therapy. Oct 2008, Vol. 15, No. 18: 1287-1293 CrossRef Recombinant adeno-associated virus-mediated human kallikrein gene therapy prevents high-salt diet-induced hypertension without effect on basal blood pressure
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