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Human Gene Therapy
Purification of Recombinant Adeno-Associated Virus Vectors by Column Chromatography and Its Performance in Vivo
To cite this article:
Guangping Gao, Guang Qu, Michael S. Burnham, James Huang, Narendra Chirmule, Bindu Joshi, Qian-Chun Yu, Jonathan A. Marsh, Christina M. Conceicao, James M. Wilson.
Human Gene Therapy.
October 2000,
11(15): 2079-2091.
doi:10.1089/104303400750001390.
Guangping Gao Institute for Human Gene Therapy and Department of Molecular and Cellular Engineering, University of Pennsylvania, Philadelphia, PA 19104 Guang Qu Institute for Human Gene Therapy and Department of Molecular and Cellular Engineering, University of Pennsylvania, Philadelphia, PA 19104 Michael S. Burnham Institute for Human Gene Therapy and Department of Molecular and Cellular Engineering, University of Pennsylvania, Philadelphia, PA 19104 James Huang Institute for Human Gene Therapy and Department of Molecular and Cellular Engineering, University of Pennsylvania, Philadelphia, PA 19104 Narendra Chirmule Institute for Human Gene Therapy and Department of Molecular and Cellular Engineering, University of Pennsylvania, Philadelphia, PA 19104 Bindu Joshi Institute for Human Gene Therapy and Department of Molecular and Cellular Engineering, University of Pennsylvania, Philadelphia, PA 19104 Qian-Chun Yu Institute for Human Gene Therapy and Department of Molecular and Cellular Engineering, University of Pennsylvania, Philadelphia, PA 19104 Jonathan A. Marsh Institute for Human Gene Therapy and Department of Molecular and Cellular Engineering, University of Pennsylvania, Philadelphia, PA 19104 Christina M. Conceicao Institute for Human Gene Therapy and Department of Molecular and Cellular Engineering, University of Pennsylvania, Philadelphia, PA 19104 James M. Wilson Institute for Human Gene Therapy and Department of Molecular and Cellular Engineering, University of Pennsylvania, Philadelphia, PA 19104 Recombinant adeno-associated virus (AAV) holds much promise for human gene therapy. While evidence indicates that AAV mediates long-term gene transfer in several different tissues, difficulty in preparing and purifying this viral vector in large quantities remains a major obstacle for evaluating AAV vectors in clinical trials. The current method of purification, based on sedimentation through cesium chloride, is not scaleable and yields product of insufficient quality. In this article we report a new technique for purifying AAV, using a fully closed two-column chromatography system. Yields of AAV vectors purified by this method are high, potency is increased, and the purity of column-purified preparations is substantially improved. We previously reported a novel method to generate AAV based on an AAV Rep/Cap-containing cell line (B50) and an Ad–AAV hybrid virus, which is amenable to scale-up in bioreactors. By combining the new, fully scaleable purification process we report here with the B50/hybrid production method, it would be feasible to prepare AAV vectors to the scale and purity required for clinical and potential commercial applications.  This paper was cited by: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. 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Jun 2008, Vol. 118, No. 5: 1955-1964 CrossRef Biochemical, Pathological, and Skeletal Improvement of Mucopolysaccharidosis VI After Gene Transfer to Liver but Not to Muscle Alessandra Tessitore, Armida Faella, Thomas O'Malley, Gabriella Cotugno, Monica Doria, Tetsuo Kunieda, Giuseppe Matarese, Mark Haskins, Alberto Auricchio Molecular Therapy. Feb 2008, Vol. 16, No. 1: 30-37 CrossRef Enhanced preparation of adeno-associated viral vectors by using high hydrostatic pressure to selectively inactivate helper adenovirus Joshua N. Leonard, Peter Ferstl, Antonio Delgado, David V. Schaffer Biotechnology and Bioengineering. Sep 2007, Vol. 97, No. 5: 1170-1179 CrossRef AP20187-Mediated Activation of A Chimeric Insulin Receptor Results in Insulin-Like Actions in Skeletal Muscle And Liver of Diabetic Mice Gabriella Cotugno, Pietro Formisano, Ferdinando Giacco, Pasqualina Colella, Francesco Beguinot, Alberto Auricchio Human Gene Therapy. Feb 2007, Vol. 18, No. 2: 106-117 Abstract | Full Text PDF | Reprints & PermissionsLong-term persistence of gene expression from adeno-associated virus serotype 5 in the mouse airways S G Sumner-Jones, L A Davies, A Varathalingam, D R Gill, S C Hyde Gene Therapy. Jan 2007, Vol. 13, No. 24: 1703-1713 CrossRef Production and characterization of adeno-associated viral vectors Joshua C Grieger, Vivian W Choi, R Jude Samulski Nature Protocols. Dec 2006, Vol. 1, No. 3: 1412-1428 CrossRef Long-Term Efficacy of Adeno-Associated Virus Serotypes 8 and 9 in Hemophilia A Dogs and Mice Rita Sarkar, Melinda Mucci, Sankar Addya, Renee Tetreault, Dwight A. Bellinger, Timothy C. Nichols, Haig H. Kazazian Jr. Human Gene Therapy. Apr 2006, Vol. 17, No. 4: 427-439 Abstract | Full Text PDF | Reprints & PermissionsLong-Term Efficacy of Adeno-associated Virus Serotypes 8 and 9 in Hemophilia A Dogs and Mice Rita Sarkar, Melinda Mucci, Sankar Addya, Renee Tetreault, Dwight A. Bellinger, Timothy C. Nichols, Haig H. Kazazian Human Gene Therapy. Mar 2006, Vol. 0, No. 0: 060217075220001 CrossRef Efficient AAV1–AAV2 Hybrid Vector for Gene Therapy of Hemophilia Bernd Hauck, Ray Ruian Xu, Jing Xie, Wenman Wu, Qiulan Ding, Matthew Sipler, Hongli Wang, Ling Chen, J. Fraser Wright, Weidong Xiao Human Gene Therapy. Jan 2006, Vol. 17, No. 1: 46-54 Abstract | Full Text PDF | Reprints & PermissionsChromatographic purification of recombinant adenoviral and adeno-associated viral vectors: methods and implications E Burova, E Ioffe Gene Therapy. Nov 2005, Vol. 12: S5-S17 CrossRef Purification of adenovirus and adeno-associated virus: comparison of novel membrane-based technology to conventional techniques A M Duffy, A M O'Doherty, T O'Brien, P M Strappe Gene Therapy. Nov 2005, Vol. 12: S62-S72 CrossRef Production of Recombinant Adeno-Associated Virus Vectors S. Zolotukhin Human Gene Therapy. May 2005, Vol. 16, No. 5: 551-557 Abstract | Full Text PDF | Reprints & PermissionsPharmacological Regulation of the Insulin Receptor Signaling Pathway Mimics Insulin Action in Cells Transduced with Viral Vectors Gabriella Cotugno, Roy Pollock, Pietro Formisano, Katja Linher, Francesco Beguinot, Alberto Auricchio Human Gene Therapy. Nov 2004, Vol. 15, No. 11: 1101-1108 Abstract | Full Text PDF | Reprints & PermissionsCharacterization of a Family of Chimpanzee Adenoviruses and Development of Molecular Clones for Gene Transfer Vectors Soumitra Roy, Guangping Gao, You Lu, Xiangyang Zhou, Martin Lock, Roberto Calcedo, James M. Wilson Human Gene Therapy. May 2004, Vol. 15, No. 5: 519-530 Abstract | Full Text PDF | Reprints & PermissionsA New Scalable Method for the Purification of Recombinant Adenovirus Vectors Anthony P. Green, James J. Huang, Miller O. Scott, Timothy D. Kierstead, Irene Beaupré, Guang-Ping Gao, James M. Wilson Human Gene Therapy. Nov 2002, Vol. 13, No. 16: 1921-1934 Abstract | Full Text PDF | Reprints & PermissionsNoninvasive gene transfer to the lung for systemic delivery of therapeutic proteins Alberto Auricchio, Erin O’Connor, Daniel Weiner, Guang-Ping Gao, Markus Hildinger, Lili Wang, Roberto Calcedo, James M. Wilson Journal of Clinical Investigation. Sep 2002, Vol. 110, No. 4: 499-504 CrossRef Muscle-Specific Promoters May Be Necessary for Adeno-Associated Virus-Mediated Gene Transfer in the Treatment of Muscular Dystrophies Laurence Cordier, Guang-Ping Gao, Andrew A. Hack, Elizabeth M. McNally, James M. Wilson, Narendra Chirmule, H. Lee Sweeney Human Gene Therapy. Jan 2001, Vol. 12, No. 2: 205-215 Abstract | Full Text PDF | Reprints & PermissionsIsolation of Highly Infectious and Pure Adeno-Associated Virus Type 2 Vectors with a Single-Step Gravity-Flow Column Alberto Auricchio, Markus Hildinger, Erin O'Connor, Guang-Ping Gao, James M. Wilson Human Gene Therapy. Jan 2001, Vol. 12, No. 1: 71-76 Abstract | Full Text PDF | Reprints & Permissions |
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