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Human Gene Therapy
Scalable Purification of Adeno-Associated Virus Type 2, 4, or 5 Using Ion-Exchange Chromatography
To cite this article:
Nikola Kaludov, Beverly Handelman, John A. Chiorini.
Human Gene Therapy.
July 2002,
13(10): 1235-1243.
doi:10.1089/104303402320139014.
Nikola Kaludov Gene Therapy and Therapeutics Branch, National Institute of Dental and Cranialfacial Research, National Institutes of Health, Bethesda, MD 20892. Beverly Handelman Gene Therapy and Therapeutics Branch, National Institute of Dental and Cranialfacial Research, National Institutes of Health, Bethesda, MD 20892. John A. Chiorini Gene Therapy and Therapeutics Branch, National Institute of Dental and Cranialfacial Research, National Institutes of Health, Bethesda, MD 20892. The availability of high-titer, high-purity, adeno-associated virus type 2 (AAV2) stocks has dramatically increased our understanding of this virus and its utility as a gene transfer vector. Current methods of purification take advantage of the stable interaction of AAV2 with heparin sulfate. This affinity chromatography, however, is not useful for purifying AAV4 and AAV5, because these serotypes lack heparin-binding activity. We have developed simple ion exchange high-performance liquid chromatography (HPLC) method for purifying different AAV serotypes that does not rely on the affinity of the viruses for heparin. The protocol is fast, efficient, and yields highly infectious material. Analysis of the highly purified virus indicated that more than 90% of the particles contained genomes and were more active than virus purified by cesium chloride (CsCl) gradient purification. This procedure is scalable and can easily be streamlined for large-scale production of recombinant adeno-associated virus (rAAV), regardless of the serotype. Ultimately, the new purification method will further the characterization of rAAV of different serotypes as vectors for gene therapy 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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Schaffer Human Gene Therapy. Apr 2007, Vol. 18, No. 4: 367-378 Abstract | Full Text PDF | Reprints & PermissionsThe use of recombinant adeno-associated virus for skeletal gene therapy Juan Dai, A. Bakr M. Rabie Orthodontics & Craniofacial Research. Mar 2007, Vol. 10, No. 1: 1-14 CrossRef Adeno-Associated Virus Serotype 2-Mediated Gene Transfer to The Parotid Glands of Nonhuman Primates Antonis Voutetakis, Changyu Zheng, Fumi Mineshiba, Ana P. Cotrim, Corinne M. Goldsmith, Michael Schmidt, Sandra Afione, Nienke Roescher, Mark Metzger, Michael A. Eckhaus, John A. Chiorini, Cynthia E. Dunbar, Robert E. Donahue, Bruce J. Baum Human Gene Therapy. Feb 2007, Vol. 18, No. 2: 142-150 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 Re-engineering Primary Epithelial Cells from Rhesus Monkey Parotid Glands for Use in Developing an Artificial Salivary Gland Simon D. Tran, Takayuki Sugito, Giovanni Dipasquale, Ana P. Cotrim, Bidhan C. Bandyopadhyay, Kathryn Riddle, David Mooney, Marc R. Kok, John A. Chiorini, Bruce J. Baum Tissue Engineering. Oct 2006, Vol. 0, No. 0: 060928130622002 CrossRef Re-engineering Primary Epithelial Cells from Rhesus Monkey Parotid Glands for Use in Developing an Artificial Salivary Gland Simon D. Tran, Takayuki Sugito, Giovanni Dipasquale, Ana P. Cotrim, Bidhan C. Bandyopadhyay, Kathryn Riddle, David Mooney, Marc R. Kok, John A. Chiorini, Bruce J. Baum Tissue Engineering. Oct 2006, Vol. 12, No. 10: 2939-2948 Abstract | Full Text PDF | Reprints & PermissionsMosaic vectors comprised of modified AAV1 capsid proteins for efficient vector purification and targeting to vascular endothelial cells M D Stachler, J S Bartlett Gene Therapy. Mar 2006 CrossRef Current issues in adeno-associated viral vector production O-W Merten, C Gény-Fiamma, A M Douar Gene Therapy. Nov 2005, Vol. 12: S51-S61 CrossRef Chromatographic 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 Recombinant Adeno-Associated Viral Vectors in the Nervous System Corinna Burger, Kevin Nash, Ronald J. Mandel Human Gene Therapy. Jul 2005, Vol. 0, No. 0: 050701034702015 CrossRef Recombinant Adeno-Associated Viral Vectors in the Nervous System Corinna Burger, Kevin Nash, Ronald J. Mandel Human Gene Therapy. Jul 2005, Vol. 16, No. 7: 781-791 Abstract | Full Text PDF | Reprints & PermissionsProduction of Recombinant Adeno-Associated Virus Vectors S. Zolotukhin Human Gene Therapy. May 2005, Vol. 16, No. 5: 551-557 Abstract | Full Text PDF | Reprints & PermissionsInfection Efficiency of Human and Mouse Embryonic Stem Cells Using Adenoviral and Adeno-Associated Viral Vectors Joseph R. Smith-Arica, Alison J. Thomson, Ray Ansell, John Chiorini, Beverly Davidson, Jim McWhir Cloning and Stem Cells. Mar 2003, Vol. 5, No. 1: 51-62 Abstract | Full Text PDF | Reprints & Permissions |
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