Manufacturing and characterizing AAV-based vectors for use in clinical studies

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine · doi:10.1038/nm1358 · Cited by 2,163

Membrane-Associated Heparan Sulfate Proteoglycan Is a Receptor for Adeno-Associated Virus Type 2 Virions

Summerford, C., & Samulski, R. J. (1998) · Journal of Virology · doi:10.1128/jvi.72.2.1438-1445.1998 · Cited by 1,409

Production of High-Titer Recombinant Adeno-Associated Virus Vectors in the Absence of Helper Adenovirus

Xiao, X., Li, J., & Samulski, R. J. (1998) · Journal of Virology · doi:10.1128/jvi.72.3.2224-2232.1998 · Cited by 1,381

Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

Zolotukhin, S., Byrne, B. J., Mason, E. G., Zolotukhin, I., Potter, M., Chesnut, K. A., Summerford, C., Samulski, R. J., & Muzyczka, N. (1999) · Gene Therapy · doi:10.1038/sj.gt.3300938 · Cited by 1,321

Use of Adeno-Associated Virus as a General Transduction Vector for Mammalian Cells

Muzyczka, N. (1992) · Current topics in microbiology and immunology · doi:10.1007/978-3-642-75608-5_5 · Cited by 756

Cd8(+) T-Cell Responses to Adeno-Associated Virus Capsid in Humans

Mingozzi, F., Maus, M. V., Hui, D. J., Sabatino, D. E., Murphy, S. L., Rasko, J. E. J., Ragni, M. V., Manno, C. S., Sommer, J., Jiang, H., et al. (2007) · Nature Medicine · doi:10.1038/nm1549 · Cited by 748

Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression

Samulski, R. J., Chang, L., & Shenk, T. E. (1989) · Journal of Virology · doi:10.1128/jvi.63.9.3822-3828.1989 · Cited by 742

Novel Tools for Production and Purification of Recombinant Adenoassociated Virus Vectors

Grimm, D., Kern, A., Rittner, K., & Kleinschmidt, J. A. (1998) · Human Gene Therapy · doi:10.1089/hum.1998.9.18-2745 · Cited by 701

Adeno-associated virus vectors can be efficiently produced without helper virus

Matsushita, T., Elliger, S., Elliger, C., Podsakoff, G., Villarreal, L., Kurtzman, G., Iwaki, Y., & Colosi, P. (1998) · Gene Therapy · doi:10.1038/sj.gt.3300680 · Cited by 507

Insect Cells as a Factory to Produce Adeno-Associated Virus Type 2 Vectors

Urabe, M., Ding, C., & Kotin, R. M. (2002) · Human Gene Therapy · doi:10.1089/10430340260355347 · Cited by 457

Infectious Clones and Vectors Derived from Adeno-Associated Virus (AAV) Serotypes Other Than AAV Type 2

Rutledge, E. A., Halbert, C. L., & Russell, D. W. (1998) · Journal of Virology · doi:10.1128/jvi.72.1.309-319.1998 · Cited by 452

A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication

Samulski, R. J., Chang, L., & Shenk, T. E. (1987) · Journal of Virology · doi:10.1128/jvi.61.10.3096-3101.1987 · Cited by 449

Binding of Adeno-associated Virus Type 5 to 2,3-Linked Sialic Acid Is Required for Gene Transfer

Walters, R. W., Yi, S. M. P., Keshavjee, S. H., Brown, K. E., Welsh, M. J., Chiorini, J. A., & Zabner, J. (2001) · Journal of Biological Chemistry · doi:10.1074/jbc.m101559200 · Cited by 362

Cloning and Characterization of Adeno-Associated Virus Type 5

Chiorini, J. A., Kim, F., Yang, L. C., & Kotin, R. M. (1999) · Journal of Virology · doi:10.1128/jvi.73.2.1309-1319.1999 · Cited by 332

Highly Purified Recombinant Adeno-Associated Virus Vectors Are Biologically Active and Free of Detectable Helper and Wild-Type Viruses

Clark, K. R., Liu, X., McGrath, J. P., & Johnson, P. R. (1999) · Human Gene Therapy · doi:10.1089/10430349950018427 · Cited by 306

Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer

Bennicelli, J., Wright, J. F., Komaromy, A., Jacobs, J. B., Hauck, B., Zelenaia, O., Mingozzi, F., Hui, D., Chung, D., Rex, T. S., et al. (2008) · Molecular Therapy · doi:10.1038/sj.mt.6300389 · Cited by 267

Cell Lines for the Production of Recombinant Adeno-Associated Virus

Clark, K. R., Voulgaropoulou, F., Fraley, D. M., & Johnson, P. R. (1995) · Human Gene Therapy · doi:10.1089/hum.1995.6.10-1329 · Cited by 257

Quantification of adeno-associated virus particles and empty capsids by optical density measurement

Sommer, J. Ü. M., Smith, P. H., Parthasarathy, S., Isaacs, J., Vijay, S., Kieran, J., Powell, S. K., McClelland, A., & Wright, J. F. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(02)00019-9 · Cited by 230

Adeno-Associated Virus Vectors in Clinical Trials

Carter, B. J. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.541 · Cited by 206

Purification of Recombinant Adeno-Associated Virus Vectors by Column Chromatography and Its Performance in Vivo

Gao, G., Qu, G., Burnham, M. S., Huang, J. J., Chirmule, N. B., Joshi, B., Yu, Q., Marsh, J. A., Conceicao, C. M., & Wilson, J. M. (2000) · Human Gene Therapy · doi:10.1089/104303400750001390 · Cited by 171

Scalable Purification of Adeno-Associated Virus Type 2, 4, or 5 Using Ion-Exchange Chromatography

Kaludov, N. K., Handelman, B., & Chiorini, J. A. (2002) · Human Gene Therapy · doi:10.1089/104303402320139014 · Cited by 167

Identification of factors that contribute to recombinant AAV2 particle aggregation and methods to prevent its occurrence during vector purification and formulation

Wright, J. F., Le, T., Prado, J., Bahr-Davidson, J., Smith, P. H., Zhen, Z., Sommer, J. M., Pierce, G. F., & Qu, G. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.02.021 · Cited by 158

Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography

Qu, G., Bahr-Davidson, J., Prado, J., Tai, A., Cataniag, F., McDonnell, J., Zhou, J.-M., Hauck, B., Luna, J., Sommer, J. M., et al. (2006) · Journal of Virological Methods · doi:10.1016/j.jviromet.2006.11.019 · Cited by 156

Treatment of human disease by adeno-associated viral gene transfer

Warrington, K. H., & Herzog, R. W. (2006) · Human Genetics · doi:10.1007/s00439-006-0165-6 · Cited by 155

High-titer recombinant adeno-associated virus production utilizing a recombinant herpes simplex virus type I vector expressing AAV-2 Rep and Cap

Conway, J. E., Rhys, C. A., Zolotukhin, I., Zolotukhin, S., Muzyczka, N., Hayward, G. S., & Byrne, B. J. (1999) · Gene Therapy · doi:10.1038/sj.gt.3300937 · Cited by 154

Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock

Davidoff, A. M., Ng, C. Y., Sleep, S., Gray, J. T., Azam, S. A., Zhao, Y., McIntosh, J., Karimipoor, M., & Nathwani, A. C. (2004) · Journal of Virological Methods · doi:10.1016/j.jviromet.2004.07.001 · Cited by 124

Identification and elimination of replication-competent adeno-associated virus (AAV) that can arise by nonhomologous recombination during AAV vector production

Allen, J. M., Debelak, D. J., Reynolds, T. C., & Miller, A. D. (1997) · Journal of Virology · doi:10.1128/jvi.71.9.6816-6822.1997 · Cited by 121

The Cutter incident: how America's first polio vaccine led to the growing vaccine crisis

(2006) · Choice Reviews Online · doi:10.5860/choice.43-2842 · Cited by 103

Evidence for Encapsidation of Prokaryotic Sequences during Recombinant Adeno-Associated Virus Production and Their in Vivo Persistence after Vector Delivery

Chadeuf, G., Ciron, C., Moullier, P., & Salvetti, A. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.06.003 · Cited by 101

Factors Affecting the Efficiency of CD8+ T Cell Cross-Priming with Exogenous Antigens

Maecker, H. T., Ghanekar, S., Suni, M. A., He, X.-S., Picker, L. J., & Maino, V. C. (2001) · The Journal of Immunology · doi:10.4049/jimmunol.166.12.7268 · Cited by 91

A Versatile and Scalable Two-Step Ion-Exchange Chromatography Process for the Purification of Recombinant Adeno-associated Virus Serotypes-2 and -5

Brument, N., Morenweiser, R., Blouin, V., Toublanc, E., Raimbaud, I., Cherel, Y., Folliot, S., Gaden, F., BOULANGER, P., KRONERLUX, G., et al. (2002) · Molecular Therapy · doi:10.1016/s1525-0016(02)90719-7 · Cited by 86

Recombinant adeno-associated virus: formulation challenges and strategies for a gene therapy vector.

Jf, W., Qu, G., Tang, C., & Sommer, J. M. (2003) · PubMed · Cited by 81

Serum-free production and column purification of adeno-associated virus type 5

Smith, R. H., Ding, C., & Kotin, R. M. (2003) · Journal of Virological Methods · doi:10.1016/j.jviromet.2003.09.002 · Cited by 69

Current status of gene therapy trials for Parkinson's disease

Fiandaca, M. S., Forsayeth, J., & Bankiewicz, K. S. (2007) · Experimental Neurology · doi:10.1016/j.expneurol.2007.08.009 · Cited by 61

Production of clinical-grade recombinant adeno-associated virus vectors

Snyder, R. O., & Flotte, T. R. (2002) · Current Opinion in Biotechnology · doi:10.1016/s0958-1669(02)00369-5 · Cited by 58

Economized large‐scale production of high yield of rAAV for gene therapy applications exploiting baculovirus expression system

Negrete, A., Yang, L. C., Mendez, A. F., Levy, J. R., & Kotin, R. M. (2007) · The Journal of Gene Medicine · doi:10.1002/jgm.1092 · Cited by 54

Infectious Titer Assay for Adeno-Associated Virus Vectors with Sensitivity Sufficient to Detect Single Infectious Events

Zhen, Z., Espinoza, Y., Bleu, T., Sommer, J. M., & Wright, J. F. (2004) · Human Gene Therapy · doi:10.1089/1043034041361262 · Cited by 51

GUIDANCE FOR INDUSTRY QUALITY SYSTEMS APPROACH TO PHARMACEUTICAL CGMP REGULATIONS

Ramya, D. S. A. S. P. (2023) · Zenodo (CERN European Organization for Nuclear Research) · doi:10.5281/zenodo.8192864 · Cited by 49

The Cutter Incident: How America’s First Polio Vaccine Led to the Growing Vaccine Crisis

Treanor, J. J. (2006) · Environmental Health Perspectives · doi:10.1289/ehp.114-a556a · Cited by 41

[24] Streamlined large-scale production of recombinant adeno-associated virus (rAAV) vectors

Potter, M., Chesnut, K. A., Muzyczka, N., Flotte, T. R., & Zolotukhin, S. (2002) · Methods in enzymology on CD-ROM/Methods in enzymology · doi:10.1016/s0076-6879(02)46069-7 · Cited by 37

Herpesvirus-based infectious titering of recombinant adeno-associated viral vectors

Mohiuddin, I. T., Loiler, S., Zolotukhin, I., Byrne, B. J., Flotte, T. R., & Snyder, R. O. (2004) · Molecular Therapy · doi:10.1016/j.ymthe.2004.08.030 · Cited by 15

900. Packaging of Host Cell and Plasmid DNA into Recombinant Adeno-Associated Virus Particles Produced by Triple Transfection

(2003) · Molecular Therapy · doi:10.1016/s1525-0016(16)41342-0 · Cited by 12

1013. In Vivo Performance of AAV2 Vectors Purified by CsCl Gradient Centrifugation or Column Chromatography

(2003) · Molecular Therapy · doi:10.1016/s1525-0016(16)41455-3 · Cited by 11

Lot Release and Characterization Testing of Live-Virus-Based Vaccines and Gene Therapy Products, Part 1 Factors Influencing Assay Choices

Gombold, J., Peden, K., Gavin, D. K., Wei, Z., Baradaran, K., Mire‐Sluis, A. R., & Schenerman, M. A. (2006) · Cited by 8

What for platelet factor 4?

Leavitt, A. D. (2007) · Blood · doi:10.1182/blood-2007-05-091363 · Cited by 6

Points to consider for aseptic processing.

(2003) · PubMed · Cited by 5

Bioseparation: Proteins

Woodard, S. L., & Nikolov, Z. L. (2010) · CRC Press eBooks · doi:10.1081/e-eafe2-120043034 · Cited by 5

Quantifying Capsid Peptide:MHC I Complexes Following Adeno-Associated Virus (AAV) Transduction

Pien, G. C., Hasbrouck, N. C., Maus, M. V., Mingozzi, F., & High, K. A. (2007) · Blood · doi:10.1182/blood.v110.11.3737.3737 · Cited by 3

Trace Capsid DNA Impurities in AAV2 Vectors Are Not Transcribed and Are Unlikely To Contribute to Capsid Peptide-MHC Class I Complexes

Hauck, B., Murphy, S. L., Lui, X., Zelenaia, O. A., High, K. A., & Wright, J. F. (2007) · Blood · doi:10.1182/blood.v110.11.3736.3736 · Cited by 2

721. Intravenous Injection Cofactors of Adeno-Associated Virus Produce Varying Tissue-Specific Transduction Levels in Adult Mice

(2007) · Molecular Therapy · doi:10.1016/s1525-0016(16)44927-0 · Cited by 1

Adeno-Associated Virus (AAV) as a Vector for Gene Therapy

Naso, M. F., Tomkowicz, B., Perry, W. L., & Strohl, W. R. (2017) · BioDrugs · doi:10.1007/s40259-017-0234-5 · Cited by 1,362

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

Mingozzi, F., & High, K. A. (2013) · Blood · doi:10.1182/blood-2013-01-306647 · Cited by 955

Emerging issues in AAV-mediated in vivo gene therapy

Colella, P., Ronzitti, G., & Mingozzi, F. (2018) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2017.11.007 · Cited by 881

Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges

Mingozzi, F., & High, K. A. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2988 · Cited by 893

Adeno-associated virus as a delivery vector for gene therapy of human diseases

Wang, J.-H., Gessler, D. J., Zhan, W., Gallagher, T. L., & Gao, G. (2024) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-024-01780-w · Cited by 904

Adeno-Associated Virus Vectorology, Manufacturing, and Clinical Applications

Grieger, J. C., & Samulski, R. J. (2012) · Methods in Enzymology · doi:10.1016/b978-0-12-386509-0.00012-0 · Cited by 199

Production of Recombinant Adeno-Associated Virus Vectors

Zolotukhin, S. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.551 · Cited by 51

Manufacturing of recombinant adeno-associated viral vectors for clinical trials

Clément, N., & Grieger, J. C. (2016) · Molecular Therapy - Methods & Clinical Development · doi:10.1038/mtm.2016.2 · Cited by 286

Transient transfection methods for clinical adeno-associated viral vector production

Wright, J. F. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.064 · Cited by 131

Quality Control Testing, Characterization and Critical Quality Attributes of Adeno‐Associated Virus Vectors Used for Human Gene Therapy

Wright, J. F. (2020) · Biotechnology Journal · doi:10.1002/biot.202000022 · Cited by 84