Manufacturing of recombinant adeno-associated viral vectors for clinical trials
Open accessAbstract
The ability to elicit robust and long-term transgene expression in vivo together with minimal immunogenicity and little to no toxicity are only a few features that make recombinant adeno-associated virus (rAAV) vectors ideally suited for many gene therapy applications. Successful preclinical studies have encouraged the use of rAAV for therapeutic gene transfer to patients in the clinical setting. Nevertheless, the use of rAAV in clinical trials has underscored the need for production and purification systems capable of generating large amounts of highly pure rAAV particles. To date, generating vector quantities sufficient to meet the expanding clinical demand is still a hurdle when using current production systems. In this chapter, we will provide a description of the current methods to produce clinical grade of rAAV under current good manufacturing practice (cGMP) settings.
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Impurities may remain in AAV preparations from the production process, such as DNA fragments from plasmids and host cell genomes as well as empty viral capsids, which can contribute to overall immunogenicity.
“In addition, impurities may remain in AAV preparations from the pro duction process, such as DNA fragments from plasmids and host cell genomes as well as empty viral capsids, which can contribute to overall immunogenicity (Wright, 2014; Cl ´ ement and Grieger, 2016; Pei et al., 2018).”
From Gene Therapy-Associated Uveitis (GTAU): Understanding and mitigating the adverse immune response in retinal gene therapy (2025) · cites this paper as Clément and Grieger, 2016
The Purification Methods for Clinical Manufacturing section (page not provided) identifies cellular nucleic acids and empty capsids as impurities that AAV purification must address. The Transfection Methods section describes plasmid-based production, but the cited passages do not specifically identify residual plasmid DNA fragments or say that these impurities contribute to immunogenicity. (LLM, conf=0.85)
Experimental — accuracy not yet measuredVirus concentration in rAAV samples covers many orders of magnitude, from 10^10 to 10^11 vg/mL at raw culture harvest to 10^13 to 10^14 vg/mL in the purified product.
“At the same time, virus concentration in samples covers many orders of magnitude, from 1010 to 1011 vg/mL at raw culture harvest to 1013 to 1014 vg/mL in the purified product,6,7,18, 19, 20,24, 25, 26, 27”
From Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies (2021) · cites this paper as [7]
Laboratory-scale production yields only a few milliliters of purified rAAV, equivalent to 10^12 to 10^14 vector genomes, such that the required sample volumes for analysis can cause significant loss of product.
“Commonly, laboratory-scale production yields only few milliliters (mL) of purified rAAV—equivalent to 1012–1014 vector genomes (vgs)—such that the required sample volumes for analysis can cause significant loss of product.6,7,20,23,24”
From Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies (2021) · cites this paper as [7]
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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 · see citing works
Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
Maguire, A. M., Simonelli, F., Pierce, E. A., Pugh, E. N., Mingozzi, F., Bennicelli, J., Banfi, S., Marshall, K. A., Testa, F., Surace, E. M., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802315 · Cited by 2,145 · see citing works
Summerford, C., & Samulski, R. J. (1998) · Journal of Virology · doi:10.1128/jvi.72.2.1438-1445.1998 · Cited by 1,409 · see citing works
Xiao, X., Li, J., & Samulski, R. J. (1998) · Journal of Virology · doi:10.1128/jvi.72.3.2224-2232.1998 · Cited by 1,381 · see citing works
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 1,317 · see citing works
Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
Nathwani, A. C., Reiss, U. M., Tuddenham, E. G. D., Rosales, C., Chowdary, P., McIntosh, J., Della Peruta, M., Lheriteau, E., Patel, N., Raj, D., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1407309 · Cited by 1,273 · see citing works
Kaplitt, M. G., Feigin, A., Tang, C., Fitzsimons, H. L., Mattis, P., Lawlor, P. A., Bland, R. J., Young, D., Strybing, K., Eidelberg, D., et al. (2007) · The Lancet · doi:10.1016/s0140-6736(07)60982-9 · Cited by 1,067 · see citing works
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 · see citing works
Maguire, A. M., High, K. A., Auricchio, A., Wright, J. F., Pierce, E. A., Testa, F., Mingozzi, F., Bennicelli, J. L., Ying, G.-S., Rossi, S., et al. (2009) · The Lancet · doi:10.1016/s0140-6736(09)61836-5 · Cited by 865 · see citing works
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
Manno, C. S., Chew, A. J., Hutchison, S., Larson, P. J., Herzog, R. W., Arruda, V. R., Tai, S. J., Ragni, M. V., Thompson, A., Ozelo, M., et al. (2003) · Blood · doi:10.1182/blood-2002-10-3296 · Cited by 749 · see citing works
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 700 · see citing works
Marks, W. J., Bartus, R. T., Siffert, J. O., Davis, C. S., Lozano, A. M., Boulis, N. M., Vitek, J. L., Stacy, M. A., Turner, D. A., Verhagen, L., et al. (2010) · The Lancet Neurology · doi:10.1016/s1474-4422(10)70254-4 · Cited by 685 · see citing works
LeWitt, P. A., Rezai, A. R., Leehey, M. A., Ojemann, S. G., Flaherty, A. W., Eskandar, E. N., Kostyk, S. K., Thomas, K., Sarkar, A., Siddiqui, M. S., et al. (2011) · The Lancet Neurology · doi:10.1016/s1474-4422(11)70039-4 · Cited by 681 · see citing works
Production and characterization of adeno-associated viral vectors
Grieger, J. C., Choi, V. W., & Samulski, R. J. (2006) · Nature Protocols · doi:10.1038/nprot.2006.207 · Cited by 567 · see citing works
Self-citationProduction and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors
Zolotukhin, S., Potter, M., Zolotukhin, I., Sakai, Y., Loiler, S., Fraites, T. J., Chiodo, V. A., Philipsberg, T., Muzyczka, N., Hauswirth, W. W., et al. (2002) · Methods · doi:10.1016/s1046-2023(02)00220-7 · Cited by 556 · see citing works
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 · see citing works
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 · see citing works
Worgall, S., Sondhi, D., Hackett, N. R., Kosofsky, B., Kekatpure, M. V., Neyzi, N., Dyke, J. P., Ballon, D., Heier, L., Greenwald, B. M., et al. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.022 · Cited by 402 · see citing works
AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
Bennett, J., Ashtari, M., Wellman, J., Marshall, K. A., Cyckowski, L. L., Chung, D. C., McCague, S., Pierce, E. A., Chen, Y., Bennicelli, J. L., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3002865 · Cited by 395 · see citing works
Nathwani, A. C., Rosales, C., McIntosh, J., Rastegarlari, G., Nathwani, D., Raj, D., Nawathe, S., Waddington, S. N., Bronson, R., Jackson, S., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2010.274 · Cited by 345 · see citing works
A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson’s disease
Muramatsu, S.-I., Fujimoto, K.-I., Kato, S., Mizukami, H., Asari, S., Ikeguchi, K., Kawakami, T., Urabe, M., Kume, A., Sato, T., et al. (2010) · Molecular Therapy · doi:10.1038/mt.2010.135 · Cited by 344 · see citing works
Flotte, T. R., Trapnell, B. C., Humphries, M., Carey, B., Calcedo, R., Rouhani, F., Campbell-Thompson, M., Yachnis, A. T., Sandhaus, R. A., McElvaney, N. G., et al. (2011) · Human Gene Therapy · doi:10.1089/hum.2011.053 · Cited by 335 · see citing works
Testa, F., Maguire, A. M., Rossi, S., Pierce, E. A., Melillo, P., Marshall, K. A., Banfi, S., Surace, E. M., Sun, J., Acerra, C., et al. (2013) · Ophthalmology · doi:10.1016/j.ophtha.2012.11.048 · Cited by 331 · see citing works
Brantly, M. L., Chulay, J. D., Wang, L., Mueller, C., Humphries, M., Spencer, L. T., Rouhani, F., Conlon, T. J., Calcedo, R., Betts, M. R., et al. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0904514106 · Cited by 327 · see citing works
Grieger, J. C., Soltys, S. M., & Samulski, R. J. (2015) · Molecular Therapy · doi:10.1038/mt.2015.187 · Cited by 326 · see citing works
Self-citationCideciyan, A. V., Hauswirth, W. W., Aleman, T. S., Kaushal, S., Schwartz, S. B., Boye, S. L., Windsor, E. A. M., Conlon, T. J., Sumaroka, A., Pang, J.-J., et al. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.086 · Cited by 312 · see citing works
Clark, K. R., Liu, X., McGrath, J. P., & Johnson, P. R. (1999) · Human Gene Therapy · doi:10.1089/10430349950018427 · Cited by 306 · see citing works
Flotte, T. R., Carter, B., Conrad, C., Guggino, W. B., Reynolds, T. C., Rosenstein, B. J., Taylor, G. R., Walden, S. M., & Wetzel, R. C. (1996) · Human Gene Therapy · doi:10.1089/hum.1996.7.9-1145 · Cited by 278 · see citing works
Tardieu, M., Zérah, M., Husson, B., de Bournonville, S., Deiva, K., Adamsbaum, C., Vincent, F., Hocquemiller, M., Broissand, C., Furlan, V., et al. (2014) · Human Gene Therapy · doi:10.1089/hum.2013.238 · Cited by 261 · see citing works
Smith, R. H., Levy, J. R., & Kotin, R. M. (2009) · Molecular Therapy · doi:10.1038/mt.2009.128 · Cited by 260 · see citing works
Gene Therapy for Aromatic <scp>l</scp> -Amino Acid Decarboxylase Deficiency
Hwu, W.-L., Muramatsu, S.-I., Tseng, S.-H., Tzen, K.-Y., Lee, N.-C., Chien, Y.-H., Snyder, R. O., Byrne, B. J., Tai, C.-H., & Wu, R.-M. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3003640 · Cited by 246 · see citing works
Hajjar, R. J., Zsebo, K. M., Deckelbaum, L. I., Thompson, C., Rudy, J., Yaroshinsky, A., Ly, H. Q., Kawase, Y., Wagner, K., Borow, K., et al. (2008) · Journal of Cardiac Failure · doi:10.1016/j.cardfail.2008.02.005 · Cited by 220 · see citing works
Adeno-Associated Virus Vectors in Clinical Trials
Carter, B. J. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.541 · Cited by 206 · see citing works
Manufacturing and characterizing AAV-based vectors for use in clinical studies
Wright, J. F. (2008) · Gene Therapy · doi:10.1038/gt.2008.65 · Cited by 205 · see citing works
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 · see citing works
Self-citationHigh-Titer Adeno-Associated Viral Vectors from a Rep/Cap Cell Line and Hybrid Shuttle Virus
Gao, G., Qu, G., Faust, L. Z., Engdahl, R. K., Xiao, W., Hughes, J. V., Zoltick, P. W., & Wilson, J. M. (1998) · Human Gene Therapy · doi:10.1089/hum.1998.9.16-2353 · Cited by 194 · see citing works
Lessons learned from the clinical development and market authorization of Glybera
Bryant, L. M., Christopher, D. M., Giles, A. R., Hinderer, C., Rodriguez, J. L., Smith, J. B., Traxler, E. A., Tycko, J., Wojno, A. P., & Wilson, J. M. (2013) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2013.087 · Cited by 189 · see citing works
Brantly, M., Spencer, L. T., Humphries, M. R., Conlon, T. J., Spencer, C. T., Poirier, A. E., Garlington, W., Baker, D. J., Song, S., Berns, K. I., et al. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.1177 · Cited by 177 · see citing works
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 · see citing works
Smith, B. K., Collins, S. W., Conlon, T. J., Mah, C. S., Lawson, L. A., Martin, A. D., Fuller, D. D., Cleaver, B. D., Clément, N., Phillips, D., et al. (2013) · Human Gene Therapy · doi:10.1089/hum.2012.250 · Cited by 158 · see citing works
Self-citationQu, 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 · see citing works
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 · see citing works
Allay, J. A., Sleep, S., Long, S., Tillman, D. M., Clark, R., Carney, G., Fagone, P., McIntosh, J. H., Nienhuis, A. W., Davidoff, A. M., et al. (2011) · Human Gene Therapy · doi:10.1089/hum.2010.202 · Cited by 152 · see citing works
Flotte, T. R., Brantly, M., Spencer, L. T., Byrne, B. J., Spencer, C. T., Baker, D. J., & Humphries, M. R. (2004) · Human Gene Therapy · doi:10.1089/10430340460732490 · Cited by 146 · see citing works
Mietzsch, M., Grasse, S., Zurawski, C., Weger, S., Bennett, A. D., Agbandje‐McKenna, M., Muzyczka, N., Zolotukhin, S., & Heilbronn, R. (2013) · Human Gene Therapy · doi:10.1089/hum.2013.184 · Cited by 141 · see citing works
Successful Production of Pseudotyped rAAV Vectors Using a Modified Baculovirus Expression System
Kohlbrenner, E., Aslanidi, G., Nash, K. R., Shklyaev, S., Campbell‐Thompson, M., Byrne, B. J., Snyder, R. O., Muzyczka, N., Warrington, K. H., & Zolotukhin, S. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.08.018 · Cited by 134 · see citing works
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 · see citing works
Gray, S. J., Choi, V. W., Asokan, A., Haberman, R. A., McCown, T. J., & Samulski, R. J. (2011) · Current Protocols in Neuroscience · doi:10.1002/0471142301.ns0417s57 · Cited by 130 · see citing works
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 · see citing works
Clément, N., Knop, D. R., & Byrne, B. J. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.094 · Cited by 119 · see citing works
Self-citationConway, J. E., Zolotukhin, S., Muzyczka, N., Hayward, G. S., & Byrne, B. J. (1997) · Journal of Virology · doi:10.1128/jvi.71.11.8780-8789.1997 · Cited by 115 · see citing works
Okada, T., Nonaka‐Sarukawa, M., Uchibori, R., Kinoshita, K., Hayashita‐Kinoh, H., Nitahara‐Kasahara, Y., Takeda, S., & Ozawa, K. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.006 · Cited by 110 · see citing works
Scalable Generation of High-Titer Recombinant Adeno-Associated Virus Type 5 in Insect Cells
Urabe, M., Nakakura, T., Xin, K.-Q., Obara, Y., Mizukami, H., Kume, A., Kotin, R. M., & Ozawa, K. (2006) · Journal of Virology · doi:10.1128/jvi.80.4.1874-1885.2006 · Cited by 110 · see citing works
Ruffing, M., Zentgraf, H., & Kleinschmidt, J. A. (1992) · Journal of Virology · doi:10.1128/jvi.66.12.6922-6930.1992 · Cited by 109 · see citing works
Martin, J. N., Wolken, N., Brown, T., Dauer, W. T., Ehrlich, M. E., & Gonzalez‐Alegre, P. (2011) · Gene Therapy · doi:10.1038/gt.2011.10 · Cited by 108 · see citing works
Koilkonda, R. D., Yu, H., Chou, T.-H., Feuer, W. J., Ruggeri, M., Porciatti, V., Tse, D., Hauswirth, W. W., Chiodo, V. A., Boye, S. L., et al. (2014) · JAMA Ophthalmology · doi:10.1001/jamaophthalmol.2013.7630 · Cited by 102 · see citing works
Current issues in adeno-associated viral vector production
Merten, O.-W., Gény-Fiamma, C., & Douar, A. M. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302615 · Cited by 101 · see citing works
Conlon, T. J., Deng, W., Erger, K. E., Cossette, T. L., Pang, J., Ryals, R. C., Clément, N., Cleaver, B. D., McDoom, I., Boye, S. L., et al. (2013) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2013.037 · Cited by 99 · see citing works
Self-citationCecchini, S., Virág, T., & Kotin, R. M. (2011) · Human Gene Therapy · doi:10.1089/hum.2010.250 · Cited by 98 · see citing works
Thomas, D. L., Wang, L., Niamke, J., Liu, J., Kang, W., Scotti, M. M., Ye, G., Veres, G., & Knop, D. R. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.004 · Cited by 97 · see citing works
Investigation of the Cause of Death in a Gene-Therapy Trial
Frank, K. M., Hogarth, D. K., Miller, J. L., Mandal, S., Mease, P. J., Samulski, R. J., Weisgerber, G. A., & Hart, J. A. L. (2009) · New England Journal of Medicine · doi:10.1056/nejmoa0801066 · Cited by 92 · see citing works
A helper virus-free packaging system for recombinant adeno-associated virus vectors
Collaco, R. F., Cao, X., & Trempe, J. P. (1999) · Gene · doi:10.1016/s0378-1119(99)00347-9 · Cited by 92 · see citing works
Herson, S., Hentati, F., Rigolet, A., Béhin, A., Romero, N. B., Leturcq, F., Laforêt, P., Maisonobe, T., Amouri, R., Haddad, H., et al. (2011) · Brain · doi:10.1093/brain/awr342 · Cited by 91 · see citing works
A Single-Step Affinity Column for Purification of Serotype-5 Based Adeno-associated Viral Vectors
Auricchio, A., O'Connor, E., Hildinger, M., & Wilson, J. M. (2001) · Molecular Therapy · doi:10.1006/mthe.2001.0462 · Cited by 91 · see citing works
Zérah, M., Piguet, F., Colle, M.-A., Raoul, S., Deschamps, J., Deniaud, J., Gautier, B., Toulgoat, F., Bièche, I., Laurendeau, I., et al. (2015) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2014.139 · Cited by 90 · see citing works
Mah, C. S., Pacak, C. A., Cresawn, K. O., DeRuisseau, L. R., Germain, S. A., Lewis, M. A., Cloutier, D. A., Fuller, D. D., & Byrne, B. J. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300100 · Cited by 90 · see citing works
Aslanidi, G., Lamb, K. E., & Zolotukhin, S. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0810614106 · Cited by 88 · see citing works
Secreted and Transmembrane Mucins Inhibit Gene Transfer with AAV4 More Efficiently than AAV5
Walters, R. W., Pilewski, J. M., Chiorini, J. A., & Zabner, J. (2002) · Journal of Biological Chemistry · doi:10.1074/jbc.m200292200 · Cited by 86 · see citing works
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 · see citing works
Stedman, H. H., Wilson, J. M., Finke, R., Kleckner, A. L., & Mendell, J. R. (2000) · Human Gene Therapy · doi:10.1089/10430340050015671 · Cited by 82 · see citing works
Martin, J. M., Frederick, A., Luo, Y., Jackson, R., Joubert, M., Sol, B., Poulin, F., Pastor, E., Armentano, D., Wadsworth, S. C., et al. (2013) · Human Gene Therapy Methods · doi:10.1089/hgtb.2013.046 · Cited by 79 · see citing works
Liu, X., Voulgaropoulou, F., Chen, R., Johnson, P. R., & Clark, K. R. (2000) · Molecular Therapy · doi:10.1006/mthe.2000.0132 · Cited by 78 · see citing works
Manufacturing Recombinant Adeno-Associated Viral Vectors from Producer Cell Clones
Thorne, B. A., Takeya, R. K., & PELUSO, R. W. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.070 · Cited by 77 · see citing works
A simplified purification protocol for recombinant adeno-associated virus vectors
Potter, M., Lins, B., Mietzsch, M., Heilbronn, R., Vliet, K. V., Chipman, P. R., Agbandje‐McKenna, M., Cleaver, B. D., Clément, N., Byrne, B. J., et al. (2014) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2014.34 · Cited by 73 · see citing works
Self-citationVirág, T., Cecchini, S., & Kotin, R. M. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.092 · Cited by 71 · see citing works
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 · see citing works
Optimized Production of High-Titer Recombinant Adeno-Associated Virus in Roller Bottles
Liu, Y.-L., Wagner, K. A., Robinson, N., Sabatino, D. E., Margaritis, P., Xiao, W., & Herzog, R. W. (2003) · BioTechniques · doi:10.2144/03341dd07 · Cited by 67 · see citing works
Chulay, J. D., Ye, G.-J., Thomas, D. L., Knop, D. R., Benson, J. M., Hutt, J. A., Wang, G., Humphries, M. R., & Flotte, T. R. (2010) · Human Gene Therapy · doi:10.1089/hum.2010.118 · Cited by 59 · see citing works
An efficient rHSV-based complementation system for the production of multiple rAAV vector serotypes
Kang, W., Wang, L., Harrell, H., Liu, J., Thomas, D., Mayfield, T. L., Scotti, M. M., Ye, G. J., Veres, G., & Knop, D. R. (2008) · Gene Therapy · doi:10.1038/gt.2008.158 · Cited by 56 · see citing works
Recent advances in recombinant adeno-associated virus vector production
Clark, K. R. (2002) · Kidney International · doi:10.1046/j.1523-1755.2002.0610s1009.x · Cited by 52 · see citing works
Flotte, T. R., Conlon, T. J., Poirier, A. E., Campbell‐Thompson, M., & Byrne, B. J. (2007) · Human Gene Therapy · doi:10.1089/hum.2006.113 · Cited by 49 · see citing works
Byrne, B. J., Smith, B. K., Mah, C. S., Lawson, L. A., Islam, S., Corti, M., Martin, D., Dobija, N., Irwin, M. V., Conlon, T. J., et al. (2014) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2014.2514 · Cited by 45 · see citing works
[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 · see citing works
Manufacturing and Regulatory Strategies for Clinical AAV2-hRPE65
Wright, J. F., Wellman, J. A., & High, K. A. (2010) · Current Gene Therapy · doi:10.2174/156652310793180715 · Cited by 36 · see citing works
Bioreactor Production of Recombinant Herpes Simplex Virus Vectors
Knop, D. R., & Harrell, H. (2007) · Biotechnology Progress · doi:10.1021/bp060373p · Cited by 31 · see citing works
Production of recombinant adeno-associated virus
Gao, G., Wilson, J. M., & Wivel, N. A. (2000) · Advances in virus research · doi:10.1016/s0065-3527(00)55016-7 · Cited by 26 · see citing works
Chiuchiolo, M. J., Kaminsky, S. M., Sondhi, D., Mancenido, D., Hollmann, C., & Crystal, R. G. (2014) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2014.2513 · Cited by 22 · see citing works
Ye, G.-J., Scotti, M. M., Thomas, D. L., Wang, L., Knop, D. R., & Chulay, J. D. (2014) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2014.060 · Cited by 21 · see citing works
Conlon, T. J., Erger, K. E., Porvasnik, S., Cossette, T. L., Roberts, C., Combee, L. A., Islam, S., Kelley, J. S., Cloutier, D., Clément, N., et al. (2013) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2013.147 · Cited by 21 · see citing works
Self-citationYe, G. J., Scotti, M. M., Liu, J., Wang, L., Knop, D. R., & Veres, G. (2010) · Gene Therapy · doi:10.1038/gt.2010.102 · Cited by 20 · see citing works
Qiu, J., Cheng, F., & Pintel, D. J. (2007) · Journal of Virology · doi:10.1128/jvi.00714-07 · Cited by 15 · see citing works
High-Yield Plasmid DNA Production
Carnes, A. E. (2012) · Genetic Engineering & Biotechnology News · doi:10.1089/gen.32.8.18 · Cited by 2 · see citing works
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Adeno-associated virus vector as a platform for gene therapy delivery
Wang, D., Tai, P. W. L., & Gao, G. (2019) · Nature Reviews Drug Discovery · doi:10.1038/s41573-019-0012-9 · Cited by 2,444 · see citing works
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 · see citing works
Gene therapy clinical trials worldwide to 2012 – an update
Ginn, S., Alexander, I. E., Edelstein, M. L., Abedi, M. R., & Wixon, J. (2013) · Journal of Gene Medicine · doi:10.1002/jgm.2698 · Cited by 807 · see citing works
Gene therapy clinical trials worldwide to 2017: An update
Ginn, S. L., Amaya, A. K., Alexander, I. E., Edelstein, M., & Abedi, M. R. (2018) · The Journal of Gene Medicine · doi:10.1002/jgm.3015 · Cited by 903 · see citing works
Adeno-associated virus as a delivery vector for gene therapy of human diseases
Wang, J.-H., Gessler, D. J., Zhan, W., Gallagher, T. F., & Gao, G. (2024) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-024-01780-w · Cited by 895 · see citing works
Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
Duan, D. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.07.011 · Cited by 489 · see citing works
The COVID-19 Vaccine Race: Challenges and Opportunities in Vaccine Formulation
Wang, J., Peng, Y., Xu, H., Cui, Z., & Williams, R. O. (2020) · AAPS PharmSciTech · doi:10.1208/s12249-020-01744-7 · Cited by 327 · see citing works
Moradi, S., Mahdizadeh, H., Šarić, T., Kim, J., Harati, J., Shahsavarani, H., Greber, B., & Moore, J. B. (2019) · Stem Cell Research & Therapy · doi:10.1186/s13287-019-1455-y · Cited by 294 · see citing works
Manufacturing challenges and rational formulation development for AAV viral vectors
Srivastava, A., Mallela, K. M. G., Deorkar, N., & Brophy, G. (2021) · Journal of Pharmaceutical Sciences · doi:10.1016/j.xphs.2021.03.024 · Cited by 269 · see citing works
HEK293 Cell Line as a Platform to Produce Recombinant Proteins and Viral Vectors
Tan, E., Chin, C. S. H., Lim, Z. F. S., & Ng, S. K. (2021) · Frontiers in Bioengineering and Biotechnology · doi:10.3389/fbioe.2021.796991 · Cited by 255 · see citing works
Bucher, K., Rodríguez-Bocanegra, E., Dauletbekov, D., & Fischer, M. D. (2021) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2020.100915 · Cited by 242 · see citing works
Pharmaceutical development of AAV-based gene therapy products for the eye
Rodrigues, G. A., Shalaev, E., Karami, T. K., Cunningham, J., Slater, N. K. H., & Rivers, H. M. (2019) · Pharmaceutical Research · doi:10.1007/s11095-018-2554-7 · Cited by 213 · see citing works
Gimpel, A. L., Katsikis, G., Sha, S., Maloney, A. J., Hong, M. S., Nguyen, T. N. T., Wolfrum, J., Springs, S. L., Sinskey, A. J., Manalis, S. R., et al. (2021) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2021.02.010 · Cited by 185 · see citing works
rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis
Shen, W., Liu, S., & Ou, L. (2022) · Frontiers in Immunology · doi:10.3389/fimmu.2022.1001263 · Cited by 173 · see citing works
Radically truncated MeCP2 rescues Rett syndrome-like neurological defects
Tillotson, R., Selfridge, J., Koerner, M. V., Gadalla, K. K. E., Guy, J., De Sousa, D., Hector, R. D., Cobb, S. R., & Bird, A. (2017) · Nature · doi:10.1038/nature24058 · Cited by 155 · see citing works
Adeno-Associated Virus Gene Therapy for Liver Disease
Kattenhorn, L. M., Tipper, C. H., Stoica, L., Geraghty, D. S., Wright, T. L., Clark, K. R., & Wadsworth, S. C. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.160 · Cited by 138 · see citing works
McIntosh, N. L., Berguig, G. Y., Karim, O. A., Cortesio, C. L., Angelis, R. D., Khan, A., Gold, D., Maga, J. A., & Bhat, V. S. (2021) · Scientific Reports · doi:10.1038/s41598-021-82599-1 · Cited by 130 · see citing works
Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy
Bailey, R. M., Armao, D. M., Kalburgi, S. N., & Gray, S. J. (2018) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.02.005 · Cited by 128 · see citing works
Ocular gene therapies in clinical practice: viral vectors and nonviral alternatives
Bordet, T., & Behar-Cohen, F. (2019) · Drug Discovery Today · doi:10.1016/j.drudis.2019.05.038 · Cited by 120 · see citing works
Werle, A. K., Powers, T. W., Zobel, J., Wappelhorst, C. N., Jarrold, M. F., Lyktey, N. A., Sloan, C. D. K., Wolf, A. J., Adams-Hall, S., Baldus, P., et al. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.08.009 · Cited by 121 · see citing works
Evolution from adherent to suspension: systems biology of HEK293 cell line development
Malm, M., Saghaleyni, R., Lundqvist, M., Giudici, M., Chotteau, V., Field, R. P., Varley, P., Hatton, D., Grassi, L., Svensson, T., et al. (2020) · Scientific Reports · doi:10.1038/s41598-020-76137-8 · Cited by 116 · see citing works
Perez, B. A., Shutterly, A., Chan, Y. K., Byrne, B. J., & Corti, M. (2020) · Brain Sciences · doi:10.3390/brainsci10020119 · Cited by 112 · see citing works
Baruteau, J., Waddington, S. N., Alexander, I. E., & Gissen, P. (2017) · Journal of Inherited Metabolic Disease · doi:10.1007/s10545-017-0053-3 · Cited by 108 · see citing works
Gadalla, K. K. E., Vudhironarit, T., Hector, R. D., Sinnett, S. E., Bahey, N. G., Bailey, M. E. S., Gray, S. J., & Cobb, S. (2017) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2017.04.007 · Cited by 109 · see citing works
Nguyen, T. N. T., Sha, S., Hong, M. S., Maloney, A. J., Barone, P. W., Neufeld, C., Wolfrum, J., Springs, S. L., Sinskey, A. J., & Braatz, R. D. (2021) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2021.04.006 · Cited by 111 · see citing works
The physico-chemical landscape of extracellular vesicles
Manno, M., Bongiovanni, A., Margolis, L. B., Bergese, P., & Arosio, P. (2024) · Nature Reviews Bioengineering · doi:10.1038/s44222-024-00255-5 · Cited by 113 · see citing works
Extension of the crRNA enhances Cpf1 gene editing in vitro and in vivo
Park, H. M., Liu, H., Wu, J., Chong, A., Mackley, V., Fellmann, C., Rao, A., Jiang, F., Chu, H., Murthy, N., et al. (2018) · Nature Communications · doi:10.1038/s41467-018-05641-3 · Cited by 102 · see citing works
Nienhuis, A. W., Nathwani, A. C., & Davidoff, A. M. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.03.033 · Cited by 99 · see citing works
Affinity chromatography for vaccines manufacturing: Finally ready for prime time?
Zhao, M., Vandersluis, M., Stout, J. G., Haupts, U. M., Sanders, M., & Jacquemart, R. (2018) · Vaccine · doi:10.1016/j.vaccine.2018.02.090 · Cited by 98 · see citing works
Production, Processing, and Characterization of Synthetic AAV Gene Therapy Vectors
Andari, J. E., & Grimm, D. (2020) · Biotechnology Journal · doi:10.1002/biot.202000025 · Cited by 97 · see citing works
Gutiérrez-Granados, S., Cervera, L., Kamen, A., & Gòdia, F. (2018) · Critical Reviews in Biotechnology · doi:10.1080/07388551.2017.1419459 · Cited by 92 · see citing works
Dobrowsky, T., Gianni, D., Pieracci, J., & Suh, J. (2021) · Current Opinion in Biomedical Engineering · doi:10.1016/j.cobme.2021.100353 · Cited by 90 · see citing works
Manufacturing of recombinant adeno-associated viruses using mammalian expression platforms
Robert, M., Chahal, P. S., Audy, A., Kamen, A., Gilbert, R., & Gaillet, B. (2017) · Biotechnology Journal · doi:10.1002/biot.201600193 · Cited by 89 · see citing works
Understanding AAV vector immunogenicity: from particle to patient
Dhungel, B. P., Winburn, I. C., Pereira, C. D. F., Huang, K., Chhabra, A., & Rasko, J. E. J. (2024) · Theranostics · doi:10.7150/thno.89380 · Cited by 90 · see citing works
High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography
Florea, M., Nicolaou, F., Pacouret, S., Zinn, E. M., Sanmiguel, J. C., Andrés‐Mateos, E., Unzu, C., Wagers, A. J., & Vandenberghe, L. H. (2022) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2022.12.009 · Cited by 88 · see citing works
Barnes, L. F., Draper, B. E., Chen, Y., Powers, T. W., & Jarrold, M. F. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.08.002 · Cited by 85 · see citing works
Bunting, S., Zhang, L., Xie, L. Y., Bullens, S., Mahimkar, R., Fong, S., Sandza, K., Harmon, D., Yates, B., Handyside, B., et al. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.12.009 · Cited by 83 · see citing works
Bailey, R. M., Rozenberg, A., & Gray, S. J. (2020) · Brain Research · doi:10.1016/j.brainres.2020.146832 · Cited by 83 · see citing works
Critical challenges and advances in recombinant adeno‐associated virus (rAAV) biomanufacturing
Fu, Q., Polanco, A., Lee, Y. S., & Yoon, S. (2023) · Biotechnology and Bioengineering · doi:10.1002/bit.28412 · Cited by 82 · see citing works
Timmers, A. M., Newmark, J. A., Turunen, H. T., Farivar, T., Liu, J., Song, C., Ye, G.-J., Pennock, S., Gaskin, C., Knop, D. R., et al. (2020) · Human Gene Therapy · doi:10.1089/hum.2019.144 · Cited by 77 · see citing works
Sinnett, S. E., Hector, R. D., Gadalla, K. K. E., Heindel, C., Chen, D., Zaric, V., Bailey, M. E. S., Cobb, S., & Gray, S. J. (2017) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2017.04.006 · Cited by 84 · see citing works
Schnödt, M., & Büning, H. (2017) · Human Gene Therapy Methods · doi:10.1089/hgtb.2016.188 · Cited by 77 · see citing works
Sinnett, S. E., Boyle, E., Lyons, C., & Gray, S. J. (2021) · Brain · doi:10.1093/brain/awab182 · Cited by 78 · see citing works
Nisanov, A. M., de Jesús, J. A. R., & Schaffer, D. V. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.03.056 · Cited by 76 · see citing works
Tran, N. T., Lecomte, E., Saleun, S., Namkung, S., Robin, C., Weber, K., Devine, E., Blouin, V., Adjali, O., Ayuso, E., et al. (2022) · Human Gene Therapy · doi:10.1089/hum.2022.050 · Cited by 74 · see citing works
An, J., Chuan-ping, Z., Qiu, H.-Y., Zhang, H., Chen, Q., Zhang, Y., Lei, X., Zhang, C., Yin, H., & Zhang, Y. (2023) · Nature Biomedical Engineering · doi:10.1038/s41551-023-01073-7 · Cited by 74 · see citing works
Molecular design for recombinant adeno-associated virus (rAAV) vector production
Aponte, J. J., Barajas, D., Peltier, J., Bardliving, C. L., Shamlou, P. A., & Gold, D. (2017) · Applied Microbiology and Biotechnology · doi:10.1007/s00253-017-8670-1 · Cited by 73 · see citing works
Adams, B., Bak, H., & Tustian, A. D. (2020) · Biotechnology and Bioengineering · doi:10.1002/bit.27472 · Cited by 72 · see citing works
Advancements in AAV-mediated Gene Therapy for Pompe Disease
Salabarria, S. M., Nair, J., Clement, N., Smith, B. K., Raben, N., Fuller, D. D., Byrne, B. J., & Corti, M. (2019) · Journal of Neuromuscular Diseases · doi:10.3233/jnd-190426 · Cited by 70 · see citing works
The membrane associated accessory protein is an adeno-associated viral egress factor
Elmore, Z. C., Havlik, L. P., Oh, D. K., Anderson, L., Daaboul, G. G., Devlin, G. W., Vincent, H. A., & Asokan, A. (2021) · Nature Communications · doi:10.1038/s41467-021-26485-4 · Cited by 70 · see citing works
Quantitative and Digital Droplet-Based AAV Genome Titration
Sanmiguel, J. C., Gao, G., & Vandenberghe, L. H. (2019) · Methods in molecular biology · doi:10.1007/978-1-4939-9139-6_4 · Cited by 69 · see citing works
Yang, H. (2025) · Pharmaceutics · doi:10.3390/pharmaceutics17081041 · Cited by 70 · see citing works
Krabbe disease successfully treated via monotherapy of intrathecal gene therapy
Bradbury, A. M., Bagel, J. H., Nguyen, D. A., Lykken, E. A., Pesayco, J. P., Jiang, X., Swain, G. P., Assenmacher, C., Hendricks, I. J., Miyadera, K., et al. (2020) · Journal of Clinical Investigation · doi:10.1172/jci133953 · Cited by 68 · see citing works
Adeno-Associated Virus Vectors: Principles, Practices, and Prospects in Gene Therapy
Zwi‐Dantsis, L., Mohamed, S., Massaro, G., & Moeendarbary, E. (2025) · Viruses · doi:10.3390/v17020239 · Cited by 63 · see citing works
Advancements in the design and scalable production of viral gene transfer vectors
Sharon, D., & Kamen, A. (2018) · Biotechnology and Bioengineering · doi:10.1002/bit.26461 · Cited by 51 · see citing works
Joshi, P. R. H., Cervera, L., Ahmed, I. A., Kondratov, O., Zolotukhin, S., Schrag, J. D., Chahal, P. S., & Kamen, A. (2019) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2019.02.003 · Cited by 60 · see citing works
Mandel, R. J., Marmion, D. J., Kirik, D., Chu, Y., Heindel, C., McCown, T., Gray, S. J., & Kordower, J. H. (2017) · Acta Neuropathologica Communications · doi:10.1186/s40478-017-0451-7 · Cited by 56 · see citing works
Development of New Strategies Using Extracellular Vesicles Loaded with Exogenous Nucleic Acid
Orefice, N. S. (2020) · Pharmaceutics · doi:10.3390/pharmaceutics12080705 · Cited by 56 · see citing works
Adeno-Associated Virus (AAV) Gene Delivery: Dissecting Molecular Interactions upon Cell Entry
Large, E. E., Silveria, M., Zane, G. M., Weerakoon, O., & Chapman, M. S. (2021) · Viruses · doi:10.3390/v13071336 · Cited by 54 · see citing works
Assessment of quality attributes for adeno‐associated viral vectors
Tustian, A. D., & Bak, H. (2021) · Biotechnology and Bioengineering · doi:10.1002/bit.27905 · Cited by 53 · see citing works
Discussing investigational AAV gene therapy with hemophilia patients: A guide
Sidonio, R. F., Pipe, S. W., Callaghan, M. U., Valentino, L. A., Monahan, P. E., & Croteau, S. E. (2020) · Blood Reviews · doi:10.1016/j.blre.2020.100759 · Cited by 52 · see citing works
Assessment of genome packaging in AAVs using Orbitrap-based charge-detection mass spectrometry
Wörner, T. P., Snijder, J., Friese, O. V., Powers, T. W., & Heck, A. J. R. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.11.013 · Cited by 51 · see citing works
Burdett, T., & Nuseibeh, S. (2022) · Gene Therapy · doi:10.1038/s41434-022-00363-0 · Cited by 51 · see citing works
Handyside, B., Ismail, A. M., Zhang, L., Yates, B., Xie, L. Y., Sihn, C., Murphy, R., Bouwman, T., Kim, C. K., Angelis, R. D., et al. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.11.001 · Cited by 49 · see citing works
Adamson-Small, L. A., Potter, M., Falk, D. J., Cleaver, B. D., Byrne, B. J., & Clément, N. (2016) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2016.31 · Cited by 51 · see citing works
Self-citationGumusgoz, E., Guisso, D. R., Kasiri, S., Wu, J., Dear, M., Verhalen, B., Nitschke, S., Mitra, S., Nitschké, F., & Minassian, B. A. (2021) · Neurotherapeutics · doi:10.1007/s13311-021-01040-7 · Cited by 50 · see citing works
Francis, J. S., Wojtas, I., Markov, V., Gray, S. J., McCown, T. J., Samulski, R. J., Bilaniuk, L. T., Wang, D.-J., De Vivo, D. C., Janson, C. G., et al. (2016) · Neurobiology of Disease · doi:10.1016/j.nbd.2016.10.001 · Cited by 49 · see citing works
An update on gene therapy for lysosomal storage disorders
Nagree, M. S., Scalia, S., McKillop, W. M., & Medin, J. A. (2019) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2019.1607837 · Cited by 50 · see citing works
Liu, S., Li, J., Peraramelli, S., Luo, N., Chen, A., Dai, M., Liu, F., Yu, Y., Leib, R. D., Li, Y., et al. (2023) · Molecular Therapy · doi:10.1016/j.ymthe.2023.11.022 · Cited by 48 · see citing works
Gene therapy for chondral and osteochondral regeneration: is the future now?
Bellavia, D., Veronesi, F., Carina, V., Costa, V., Raimondi, L., Luca, A. D., Alessandro, R., Fini, M., & Giavaresi, G. (2017) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-017-2637-3 · Cited by 48 · see citing works
Target-Cell-Directed Bioengineering Approaches for Gene Therapy of Hemophilia A
Brown, H. C., Zakas, P. M., George, S., Parker, E. T., Spencer, H. T., & Doering, C. B. (2018) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.01.004 · Cited by 45 · see citing works
Le, D. T., Radukic, M. T., & Müller, K. M. (2019) · Scientific Reports · doi:10.1038/s41598-019-54928-y · Cited by 45 · see citing works
Fong, S., Handyside, B., Sihn, C., Liu, S., Zhang, L., Xie, L. Y., Murphy, R., Galicia, N., Yates, B., Minto, W. C., et al. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.07.005 · Cited by 43 · see citing works
Translating CRISPR-Cas Therapeutics: Approaches and Challenges
Tay, L. S., Palmer, N. D., Panwala, R., Chew, W. L., & Mali, P. (2020) · The CRISPR Journal · doi:10.1089/crispr.2020.0025 · Cited by 43 · see citing works
Judson, M. C., Shyng, C., Simon, J. M., Davis, C. R., Punt, A. M., Salmon, M. T., Miller, N. W., Ritola, K., Elgersma, Y., Amaral, D. G., et al. (2021) · JCI Insight · doi:10.1172/jci.insight.144712 · Cited by 45 · see citing works
Lessons learned from lung and liver in-vivo gene therapy: implications for the future
van Haasteren, J., Hyde, S. C., & Gill, D. R. (2018) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2018.1506761 · Cited by 40 · see citing works
Construction of an rAAV Producer Cell Line through Synthetic Biology
Lee, Z., Lu, M., Irfanullah, E., Soukup, M., & Hu, W. (2022) · ACS Synthetic Biology · doi:10.1021/acssynbio.2c00207 · Cited by 40 · see citing works
State‐of‐the‐art 2023 on gene therapy for phenylketonuria
Martinez, M., Harding, C. O., Schwank, G., & Thöny, B. (2023) · Journal of Inherited Metabolic Disease · doi:10.1002/jimd.12651 · Cited by 38 · see citing works
Stowaways in the cargo: Contaminating nucleic acids in rAAV preparations for gene therapy
Brimble, M. A., Winston, S. M., & Davidoff, A. M. (2023) · Molecular Therapy · doi:10.1016/j.ymthe.2023.07.025 · Cited by 39 · see citing works
Zhang, C.-X., & Meagher, M. M. (2017) · Analytical Chemistry · doi:10.1021/acs.analchem.6b02933 · Cited by 39 · see citing works
Treatment to cure: Advancing AAV gene therapy manufacture
Young, P. M. (2023) · Drug Discovery Today · doi:10.1016/j.drudis.2023.103610 · Cited by 38 · see citing works
Marichal‐Gallardo, P., Börner, K., Pieler, M., Sonntag-Buck, V., Obr, M., Bejarano, D. A., Wolff, M. W., Kräusslich, H., Reichl, U., & Grimm, D. (2021) · Human Gene Therapy · doi:10.1089/hum.2019.284 · Cited by 38 · see citing works
Su, W., Patrício, M. I., Duffy, M. R., Krakowiak, J. M., Seymour, L. W., & Cawood, R. (2022) · Nature Communications · doi:10.1038/s41467-022-28738-2 · Cited by 40 · see citing works
The ice age – A review on formulation of Adeno-associated virus therapeutics
Grossen, P., Skaripa-Koukelli, I., van Haasteren, J., Machado, A. H. E., & Dürr, C. (2023) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2023.07.002 · Cited by 36 · see citing works
A Robust System for Production of Superabundant VP1 Recombinant AAV Vectors
Qizhao, W., Wu, Z., Zhang, J., Firrman, J., Wei, H., Zhuang, Z., Liu, L., Miao, L., Hu, Y., Li, D., et al. (2017) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2017.11.002 · Cited by 37 · see citing works
Nucleic acid-based therapeutics for dermal wound healing
Sharma, P. M., Kumar, A., Agarwal, T., Dey, A. D., Moghaddam, F. D., Rahimmanesh, I., Ghovvati, M., Yousefiasl, S., Borzacchiello, A., Mohammadi, A., et al. (2022) · International Journal of Biological Macromolecules · doi:10.1016/j.ijbiomac.2022.08.099 · Cited by 36 · see citing works
Adamson-Small, L. A., Potter, M., Byrne, B. J., & Clément, N. (2017) · Human Gene Therapy Methods · doi:10.1089/hgtb.2016.151 · Cited by 35 · see citing works
Self-citationLing, Q., Rioux, M., Hu, Y., Lee, M., & Gray, S. J. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.09.001 · Cited by 34 · see citing works
Peptide ligands for the affinity purification of adeno‐associated viruses from HEK 293 cell lysates
Chu, W., Shastry, S., Barbieri, E., Prodromou, R., Greback‐Clarke, P., Smith, W. K., Moore, B., Kilgore, R., Cummings, C., Pancorbo, J., et al. (2023) · Biotechnology and Bioengineering · doi:10.1002/bit.28495 · Cited by 33 · see citing works
Kurasawa, J. H., PARK, A. W., Sowers, C. R., Halpin, R., Tovchigrechko, A., Dobson, C., Schmelzer, A. E., Gao, C., Wilson, S. D., & Ikeda, Y. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.09.018 · Cited by 34 · see citing works
Chimeric Adeno-Associated Virus-Mediated Cardiovascular Reprogramming for Ischemic Heart Disease
Yoo, S. Y., Jeong, S.-N., Kang, J., & Lee, S. (2018) · ACS Omega · doi:10.1021/acsomega.8b00904 · Cited by 33 · see citing works
Warriner, L. W., Duke, J. R., Pack, D. W., & DeRouchey, J. E. (2018) · Biomacromolecules · doi:10.1021/acs.biomac.8b01248 · Cited by 32 · see citing works
Bracewell, D. G., Smith, V., Delahaye, M., & Smales, C. M. (2021) · Current Opinion in Biotechnology · doi:10.1016/j.copbio.2021.06.026 · Cited by 32 · see citing works
Gumusgoz, E., Kasiri, S., Guisso, D. R., Wu, J., Dear, M., Verhalen, B., & Minassian, B. A. (2022) · Neurotherapeutics · doi:10.1007/s13311-022-01218-7 · Cited by 31 · see citing works
Reddy, J. V., Raudenbush, K., Papoutsakis, E. T., & Ierapetritou, M. (2023) · Biotechnology Advances · doi:10.1016/j.biotechadv.2023.108179 · Cited by 32 · see citing works
Selvaraj, N., Wang, C.-K., Bowser, B., Broadt, T. L., Shaban, S., Burns, J., Saptharishi, N., Pechan, P. A., Golebiowski, D., Alimardanov, A., et al. (2021) · Human Gene Therapy · doi:10.1089/hum.2020.054 · Cited by 29 · see citing works
Fakhiri, J., & Grimm, D. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.04.005 · Cited by 29 · see citing works
Clérin, E., Marussig, M., Sahel, J., & Léveillard, T. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21051625 · Cited by 27 · see citing works
Genome-wide activation screens to increase adeno-associated virus production
Barnes, C. R., Lee, H., Ojala, D. S., Lewis, K. K., Limsirichai, P., & Schaffer, D. V. (2021) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2021.06.026 · Cited by 28 · see citing works
Gene therapy: challenges in cell culture scale-up
Shupe, J., Zhang, A., Odenwelder, D. C., & Dobrowsky, T. M. (2022) · Current Opinion in Biotechnology · doi:10.1016/j.copbio.2022.102721 · Cited by 28 · see citing works
- Authors
- Clément, N., & Grieger, J. C.
- Affiliations
- University of Florida (US)
- Corresponding author
- Grieger, Joshua C
- Venue
- Molecular Therapy - Methods & Clinical Development
- Publisher
- Elsevier BV
- Published
- 2016
- Volume
- 3
- Pages
- 16002
- ISSN
- 2329-0501 (ISSN-L)
- PMC
- PMC4804725
- OpenAlex
- W2324542890
- Cited by
- 286
- References
- 93
- Citations by year
- 2026: 19 · 2025: 31 · 2024: 38 · 2023: 31 · 2022: 25 · 2021: 33 · 2020: 33 · 2019: 19 · 2018: 23 · 2017: 26 · 2016: 7 · 2013: 1
- FWCI
- 3.34
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Molecular Therapy — Methods & Clinical Development (publishedVersion, cc-by-nc-nd)
- Europe PMC (PubMed Central) (publishedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Viral Infectious Diseases and Gene Expression in Insects
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- recombinant adeno-associated virus vectors, AAV vector production, vector purification, current good manufacturing practice, gene therapy, therapeutic gene transfer, clinical trials
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