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

The amyloid hypothesis of Alzheimer's disease at 25 years

Selkoe, D. J., & Hardy, J. (2016) · EMBO Molecular Medicine · doi:10.15252/emmm.201606210 · Cited by 6,164 · see citing works

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

Zincarelli, C., Soltys, S., Rengo, G., & Rabinowitz, J. E. (2008) · Molecular Therapy · doi:10.1038/mt.2008.76 · Cited by 1,490 · see citing works

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 · see citing works

Clades of Adeno-Associated Viruses Are Widely Disseminated in Human Tissues

Gao, G., Vandenberghe, L. H., Alvira, M. R., Lu, Y., Calcedo, R., Zhou, X., & Wilson, J. M. (2004) · Journal of Virology · doi:10.1128/jvi.78.12.6381-6388.2004 · Cited by 1,070 · see citing works

Effect of genome size on AAV vector packaging

Wu, Z., Yang, H., & Colosi, P. (2010) · Molecular Therapy · doi:10.1038/mt.2009.255 · Cited by 1,025 · 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

Adeno-associated Virus Serotypes: Vector Toolkit for Human Gene Therapy

Wu, Z., Asokan, A., & Samulski, R. J. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.05.009 · Cited by 921 · see citing works

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 · see citing works

Codon Bias as a Means to Fine-Tune Gene Expression

Quax, T. E. F., Claassens, N. J., Söll, D. G., & John van der, O. O. S. T. (2015) · Molecular Cell · doi:10.1016/j.molcel.2015.05.035 · Cited by 779 · see citing works

Engineering adeno-associated viruses for clinical gene therapy

Kotterman, M. A., & Schaffer, D. V. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3742 · Cited by 761 · see citing works

Recombinant AAV Viral Vectors Pseudotyped with Viral Capsids from Serotypes 1, 2, and 5 Display Differential Efficiency and Cell Tropism after Delivery to Different Regions of the Central Nervous System

Burger, C., Gorbatyuk, O. S., Velardo, M. J., Peden, C. S., Williams, P., Zolotukhin, S., Reier, P. J., Mandel, R. J., & Muzyczka, N. (2004) · Molecular Therapy · doi:10.1016/j.ymthe.2004.05.024 · Cited by 741 · see citing works

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

McCarty, D., Monahan, P., & Samulski, R. (2001) · Gene Therapy · doi:10.1038/sj.gt.3301514 · Cited by 735 · see citing works

In Vivo–Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous

Dalkara, D., Byrne, L. C., Klimczak, R. R., Visel, M., Yin, L., Merigan, W. H., Flannery, J. G., & Schaffer, D. V. (2013) · Science Translational Medicine · doi:10.1126/scitranslmed.3005708 · Cited by 734 · cited 10× · see citing works

From the Cover: Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system

Davidson, B. L., Stein, C. S., Heth, J., Martins, I., Kotin, R. M., Derksen, T. A., Zabner, J., Ghodsi, A., & Chiorini, J. A. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.050581197 · Cited by 679 · see citing works

Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart

Wang, Z., Zhu, T., Qiao, C., Zhou, L., Wang, B., Zhang, J., Chen, C., Li, J., & Xiao, X. (2005) · Nature Biotechnology · doi:10.1038/nbt1073 · Cited by 602 · see citing works

AAV-Mediated Gene Therapy for Research and Therapeutic Purposes

Samulski, R. J., & Muzyczka, N. (2014) · Annual Review of Virology · doi:10.1146/annurev-virology-031413-085355 · Cited by 582 · see citing works

Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses

Zhong, L., Li, B., Mah, C. S., Govindasamy, L., Agbandje-McKenna, M., Cooper, M., Herzog, R. W., Zolotukhin, I., Warrington, K. H., Weigel-Van Aken, K. A., et al. (2008) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0802866105 · Cited by 558 · see citing works

Gene therapy vectors based on adeno-associated virus type 1

Xiao, W., Chirmule, N., Berta, S. C., McCullough, B., Gao, G., & Wilson, J. M. (1999) · Journal of Virology · doi:10.1128/jvi.73.5.3994-4003.1999 · Cited by 541 · see citing works

An essential receptor for adeno-associated virus infection

Pillay, S., Meyer, N. L., Puschnik, A. S., Davulcu, O., Diep, J., Ishikawa, Y., Jae, L. T., Wosen, J. E., Nagamine, C. M., Chapman, M. S., et al. (2016) · Nature · doi:10.1038/nature16465 · Cited by 532 · see citing works

Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system

Davidson, B. L., Stein, C. S., Heth, J. A., Martins, I., Kotin, R. M., Derksen, T. A., Zabner, J., Ghodsi, A., & Chiorini, J. A. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.97.7.3428 · Cited by 531 · see citing works

Calcium upregulation by percutaneous administration of gene therapy in patients with cardiac disease (CUPID 2): a randomised, multinational, double-blind, placebo-controlled, phase 2b trial

Greenberg, B., Butler, J., Felker, G. M., Ponikowski, P., Voors, A. A., Desai, A. S., Barnard, D., Bouchard, A., Jaski, B., Lyon, A. R., et al. (2016) · The Lancet · doi:10.1016/s0140-6736(16)00082-9 · Cited by 525 · cited 1× · see citing works

Self-complementary AAV vectors; advances and applications

McCarty, D. M. (2008) · Molecular Therapy · doi:10.1038/mt.2008.171 · Cited by 515 · see citing works

Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas

Nault, J.-C., Datta, S., Imbeaud, S., Franconi, A., Mallet, M., Couchy, G., Letouzé, E., Pilati, C., Verret, B., Blanc, J.-F., et al. (2015) · Nature Genetics · doi:10.1038/ng.3389 · Cited by 489 · see citing works

The AAV Vector Toolkit: Poised at the Clinical Crossroads

Asokan, A., Schaffer, D. V., & Samulski, R. J. (2012) · Molecular Therapy · doi:10.1038/mt.2011.287 · Cited by 463 · see citing works

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

Bennett, J., Wellman, J., Marshall, K. A., McCague, S., Ashtari, M., DiStefano-Pappas, J., Elci, O. U., Chung, D. C., Sun, J., Wright, J. F., et al. (2016) · The Lancet · doi:10.1016/s0140-6736(16)30371-3 · Cited by 461 · see citing works

Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2

Qing, K., Mah, C., Hansen, J., Zhou, S., Dwarki, V., & Srivastava, A. (1999) · Nature Medicine · doi:10.1038/4758 · Cited by 443 · see citing works

The 37/67-Kilodalton Laminin Receptor Is a Receptor for Adeno-Associated Virus Serotypes 8, 2, 3, and 9

Akache, B., Grimm, D., Pandey, K., Yant, S. R., Xu, H., & Kay, M. A. (2006) · Journal of Virology · doi:10.1128/jvi.00878-06 · Cited by 430 · see citing works

Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors

Gray, S. J., Foti, S. B., Schwartz, J. W., Bachaboina, L., Taylor-Blake, B., Coleman, J., Ehlers, M. D., Zylka, M. J., McCown, T. J., & Samulski, R. J. (2011) · Human Gene Therapy · doi:10.1089/hum.2010.245 · Cited by 420 · see citing works

AAV serotype 2 vectors preferentially integrate into active genes in mice

Nakai, H., Montini, E., Fuess, S., Storm, T. A., Grompe, M., & Kay, M. A. (2003) · Nature Genetics · doi:10.1038/ng1179 · Cited by 392 · see citing works

Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus

Duan, D., Yue, Y., Yan, Z., Yang, J., & Engelhardt, J. F. (2000) · Journal of Clinical Investigation · doi:10.1172/jci8317 · Cited by 391 · see citing works

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

Gray, S. J., Nagabhushan Kalburgi, S., McCown, T. J., & Jude Samulski, R. (2013) · Gene Therapy · doi:10.1038/gt.2012.101 · Cited by 377 · see citing works

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 360 · see citing works

Pre-existing Anti–Adeno-Associated Virus Antibodies as a Challenge in AAV Gene Therapy

Louis Jeune, V., Joergensen, J. A., Hajjar, R. J., & Weber, T. (2013) · Human Gene Therapy Methods · doi:10.1089/hgtb.2012.243 · Cited by 358 · see citing works

Identification of PDGFR as a receptor for AAV-5 transduction

Pasquale, G. D., Davidson, B. L., Stein, C. S., Martins, I., Scudiero, D., Monks, A., & Chiorini, J. A. (2003) · Nature Medicine · doi:10.1038/nm929 · Cited by 355 · see citing works

Three-Year Follow-up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2

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

Rapid, Simple, and Versatile Manufacturing of Recombinant Adeno-Associated Viral Vectors at Scale

Lock, M. W., Alvira, M. R., Vandenberghe, L. H., Samanta, A., Toelen, J., Debyser, Z., & Wilson, J. M. (2010) · Human Gene Therapy · doi:10.1089/hum.2010.055 · Cited by 328 · see citing works

α2,3 and α2,6 N-Linked Sialic Acids Facilitate Efficient Binding and Transduction by Adeno-Associated Virus Types 1 and 6

Wu, Z., Miller, E. B., Agbandje‐McKenna, M., & Samulski, R. J. (2006) · Journal of Virology · doi:10.1128/jvi.00895-06 · Cited by 327 · see citing works

Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector

Grieger, J. C., Soltys, S. M., & Samulski, R. J. (2015) · Molecular Therapy · doi:10.1038/mt.2015.187 · Cited by 326 · see citing works

Unrestricted Hepatocyte Transduction with Adeno-Associated Virus Serotype 8 Vectors in Mice

Nakai, H., Fuess, S., Storm, T. A., Muramatsu, S., Nara, Y., & Kay, M. A. (2004) · Journal of Virology · doi:10.1128/jvi.79.1.214-224.2005 · Cited by 322 · see citing works

Adeno-Associated Virus (AAV) Serotype 9 Provides Global Cardiac Gene Transfer Superior to AAV1, AAV6, AAV7, and AAV8 in the Mouse and Rat

Bish, L. T., Morine, K., Sleeper, M. M., Sanmiguel, J. C., Wu, D., Gao, G., Wilson, J. M., & Sweeney, H. L. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.123 · Cited by 291 · see citing works

Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System

Zhang, H., Yang, B., Mu, X., Ahmed, S. S., Su, Q., He, R., Wang, H., Mueller, C., Sena-Esteves, M., Brown, R., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2011.98 · Cited by 291 · see citing works

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Hocquemiller, M., Giersch, L., Audrain, M., Parker, S., & Cartier, N. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.087 · Cited by 291 · see citing works

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 · see citing works

Adeno-Associated Virus at 50: A Golden Anniversary of Discovery, Research, and Gene Therapy Success—A Personal Perspective

Hastie, E. L., & Samulski, R. J. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.025 · Cited by 284 · see citing works

Terminal N-Linked Galactose Is the Primary Receptor for Adeno-associated Virus 9

Shen, S., Bryant, K. D., Brown, S. M., Randell, S. H., & Asokan, A. (2011) · Journal of Biological Chemistry · doi:10.1074/jbc.m110.210922 · Cited by 281 · see citing works

Production and clinical development of nanoparticles for gene delivery

Chen, J., Guo, Z., Tian, H., & Chen, X. (2016) · Molecular Therapy - Methods & Clinical Development · doi:10.1038/mtm.2016.23 · Cited by 274 · see citing works

A Simplified Baculovirus-AAV Expression Vector System Coupled With One-step Affinity Purification Yields High-titer rAAV Stocks From Insect Cells

Smith, R. H., Levy, J. R., & Kotin, R. M. (2009) · Molecular Therapy · doi:10.1038/mt.2009.128 · Cited by 260 · see citing works

Intracellular transport of recombinant adeno-associated virus vectors

Nonnenmacher, M., & Weber, T. (2012) · Gene Therapy · doi:10.1038/gt.2012.6 · Cited by 258 · see citing works

Structure of Adeno-Associated Virus Serotype 8, a Gene Therapy Vector

Nam, H.-J., Lane, M. D., Padron, E., Gurda, B. L., McKENNA, R. V., Kohlbrenner, E., Aslanidi, G., Byrne, B. J., Muzyczka, N., Zolotukhin, S., et al. (2007) · Journal of Virology · doi:10.1128/jvi.01304-07 · Cited by 256 · see citing works

CpG-depleted adeno-associated virus vectors evade immune detection

Faust, S. M., Bell, P., Cutler, B. J., Ashley, S. N., Zhu, Y., Rabinowitz, J. E., & Wilson, J. M. (2013) · Journal of Clinical Investigation · doi:10.1172/jci68205 · Cited by 254 · see citing works

Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6

Blankinship, M. J., Gregorevic, P., Allen, J. M., Harper, S. Q., Harper, H. A., Halbert, C. L., Miller, A. D., & Chamberlain, J. S. (2004) · Molecular Therapy · doi:10.1016/j.ymthe.2004.07.016 · Cited by 250 · see citing works

Inclusion of the Hepatic Locus Control Region, an Intron, and Untranslated Region Increases and Stabilizes Hepatic Factor IX Gene Expression in Vivo but Not in Vitro

Miao, C. H., Ohashi, K., Patijn, G. A., Meuse, L. A., Ye, X., Thompson, A. R., & Kay, M. A. (2000) · Molecular Therapy · doi:10.1006/mthe.2000.0075 · Cited by 246 · see citing works

Hepatocyte Growth Factor Receptor Is a Coreceptor for Adeno-Associated Virus Type 2 Infection

Kashiwakura, Y., Tamayose, K., Iwabuchi, K., Hirai, Y., Shimada, T., Matsumoto, K., Nakamura, T., Watanabe, M., Oshimi, K., & Daida, H. (2005) · Journal of Virology · doi:10.1128/jvi.79.1.609-614.2005 · Cited by 246 · see citing works

Tissue-specific codon usage and the expression of human genes

Plotkin, J. B., Robins, H. S., & Levine, A. J. (2004) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0404957101 · Cited by 235 · see citing works

Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression

Zhong, L., Li, B., Jayandharan, G., Mah, C. S., Govindasamy, L., Agbandje-McKenna, M., Herzog, R. W., Weigel-Van Aken, K. A., Hobbs, J. A., Zolotukhin, S., et al. (2008) · Virology · doi:10.1016/j.virol.2008.08.027 · Cited by 232 · see citing works

Characterization of Genome Integrity for Oversized Recombinant AAV Vector

Dong, B., Nakai, H., & Xiao, W. (2010) · Molecular Therapy · doi:10.1038/mt.2009.258 · Cited by 230 · see citing works

Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy

Balakrishnan, B., & Jayandharan, G. (2014) · Current Gene Therapy · doi:10.2174/1566523214666140302193709 · Cited by 225 · see citing works

Structural insight into the unique properties of adeno-associated virus serotype 9

DiMattia, M. A., Nam, H.-J., Van Vliet, K., Mitchell, M., Bennett, A., Gurda, B. L., McKenna, R., Olson, N. H., Sinkovits, R. S., Potter, M., et al. (2012) · Journal of Virology · doi:10.1128/jvi.07232-11 · Cited by 219 · see citing works

AAV Capsid Structure and Cell Interactions

Agbandje-McKenna, M., & Kleinschmidt, J. (2012) · Methods in Molecular Biology · doi:10.1007/978-1-61779-370-7_3 · Cited by 215 · see citing works

Global CNS Transduction of Adult Mice by Intravenously Delivered rAAVrh.8 and rAAVrh.10 and Nonhuman Primates by rAAVrh.10

Yang, B., Li, S., Wang, H., Guo, Y., Gessler, D. J., Cao, C., Su, Q., Kramer, J., Zhong, L., Ahmed, S. S., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.68 · Cited by 213 · see citing works

Adeno-Associated Virus Type 2 VP2 Capsid Protein Is Nonessential and Can Tolerate Large Peptide Insertions at Its N Terminus

Warrington, K. H., Gorbatyuk, O. S., Harrison, J. K., Opie, S. R., Zolotukhin, S., & Muzyczka, N. (2004) · Journal of Virology · doi:10.1128/jvi.78.12.6595-6609.2004 · Cited by 205 · 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

Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models

Chandler, R. J., Sands, M. S., & Venditti, C. P. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.009 · Cited by 203 · see citing works

Gene therapy for the CNS using AAVs: The impact of systemic delivery by AAV9

Saraiva, J., Nobre, R. J., & Pereira de Almeida, L. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.09.011 · Cited by 201 · see citing works

Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors

Pien, G. C., Basner-Tschakarjan, E., Hui, D. J., Mentlik, A. N., Finn, J. D., Hasbrouck, N. C., Zhou, S., Murphy, S. L., Maus, M. V., Mingozzi, F., et al. (2009) · Journal of Clinical Investigation · doi:10.1172/jci36891 · Cited by 192 · see citing works

Adeno-Associated Virus Type 2 Contains an Integrin α5β1 Binding Domain Essential for Viral Cell Entry

Asokan, A., Hamra, J. B., Govindasamy, L., Agbandje-McKenna, M., & Samulski, R. J. (2006) · Journal of Virology · doi:10.1128/jvi.00843-06 · Cited by 183 · see citing works

Transduction Efficiencies of Novel AAV Vectors in Mouse Airway Epithelium In Vivo and Human Ciliated Airway Epithelium In Vitro

Limberis, M. P., Vandenberghe, L. H., Zhang, L., Pickles, R. J., & Wilson, J. M. (2008) · Molecular Therapy · doi:10.1038/mt.2008.261 · Cited by 180 · see citing works

Displaying High-affinity Ligands on Adeno-associated Viral Vectors Enables Tumor Cell-specific and Safe Gene Transfer

Münch, R. C., Janicki, H., Völker, I., Rasbach, A., Hallek, M. J., Büning, H., & Buchholz, C. J. (2012) · Molecular Therapy · doi:10.1038/mt.2012.186 · Cited by 176 · see citing works

Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes

Xu, R., Janson, C., Mastakov, M., Lawlor, P., Young, D., Mouravlev, A., Fitzsimons, H., Choi, K.-L., Ma, H., Dragunow, M., et al. (2001) · Gene Therapy · doi:10.1038/sj.gt.3301529 · Cited by 174 · see citing works

Directed evolution of novel adeno-associated viruses for therapeutic gene delivery

Bartel, M. A., Weinstein, J. R., & Schaffer, D. V. (2012) · Gene Therapy · doi:10.1038/gt.2012.20 · Cited by 162 · see citing works

Comparative Analysis of Adeno-Associated Virus Capsid Stability and Dynamics

Rayaprolu, V., Kruse, S., Kant, R., Venkatakrishnan, B., Movahed, N., Brooke, D. J., Lins, B., Bennett, A. D., Potter, T., McKENNA, R. V., et al. (2013) · Journal of Virology · doi:10.1128/jvi.01415-13 · Cited by 161 · see citing works

Design of a Phase 2b Trial of Intracoronary Administration of AAV1/SERCA2a in Patients With Advanced Heart Failure

Greenberg, B., Yaroshinsky, A., Zsebo, K. M., Butler, J., Felker, G. M., Voors, A. A., Rudy, J. J., Wagner, K., & Hajjar, R. J. (2014) · JACC Heart Failure · doi:10.1016/j.jchf.2013.09.008 · Cited by 159 · see citing works

Production, purification and characterization of adeno-associated vectors

Ayuso, E., Mingozzi, F., & Bosch, F. (2010) · Current Gene Therapy · doi:10.2174/156652310793797685 · Cited by 151 · see citing works

Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids

Castle, M. J., Turunen, H. T., Vandenberghe, L. H., & Wolfe, J. H. (2016) · Methods in Molecular Biology · doi:10.1007/978-1-4939-3271-9_10 · Cited by 151 · cited 10× · see citing works

Construction and analysis of compact muscle-specific promoters for AAV vectors

Wang, B., Li, J., Fu, F. H., Chen, C., Zhu, X., Zhou, L., Jiang, X. L., & Xiao, X. (2008) · Gene Therapy · doi:10.1038/gt.2008.104 · Cited by 149 · see citing works

Host Cell DNA Repair Pathways in Adeno-Associated Viral Genome Processing

Choi, V. W., McCarty, D. M., & Samulski, R. J. (2006) · Journal of Virology · doi:10.1128/jvi.00841-06 · Cited by 144 · see citing works

Viral Expression Cassette Elements to Enhance Transgene Target Specificity and Expression in Gene Therapy

The University of North Carolina at Chapel Hill University Libraries (2020) · PubMed · doi:10.17615/2x9m-k248 · Cited by 144 · see citing works

Structural Characterization of the Dual Glycan Binding Adeno-Associated Virus Serotype 6

Ng, R., Lakshmanan Govindasamy, A., Gurda, B. L., McKENNA, R. V., Kozyreva, O. G., Samulski, R. J., Parent, K. N., Baker, T. S., & Agbandje‐McKenna, M. (2010) · Journal of Virology · doi:10.1128/jvi.01235-10 · Cited by 143 · see citing works

OneBac: Platform for Scalable and High-Titer Production of Adeno-Associated Virus Serotype 1–12 Vectors for Gene Therapy

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

Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids

Chamberlain, K., Riyad, J. M., & Weber, T. (2016) · Human Gene Therapy Methods · doi:10.1089/hgtb.2015.140 · Cited by 141 · cited 1× · see citing works

Recombinant AAV serotype 1 transduction efficiency and tropism in the murine brain

Wang, C., Wang, C.-M., Clark, K. R., & Sferra, T. J. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302011 · Cited by 139 · see citing works

Identification of the Galactose Binding Domain of the Adeno-Associated Virus Serotype 9 Capsid

Bell, C. L., Gurda, B. L., Vliet, K. V., Agbandje‐McKenna, M., & Wilson, J. M. (2012) · Journal of Virology · doi:10.1128/jvi.00448-12 · Cited by 138 · 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

Adeno-Associated Virus Vectors and Neurological Gene Therapy

Ojala, D. S., Amara, D. P., & Schaffer, D. V. (2014) · The Neuroscientist · doi:10.1177/1073858414521870 · Cited by 137 · see citing works

Mapping the AAV Capsid Host Antibody Response toward the Development of Second Generation Gene Delivery Vectors

Tseng, Y.-S., & Agbandje‐McKenna, M. (2014) · Frontiers in Immunology · doi:10.3389/fimmu.2014.00009 · Cited by 134 · see citing works

Adeno-Associated Virus Capsids Displaying Immunoglobulin-Binding Domains Permit Antibody-Mediated Vector Retargeting to Specific Cell Surface Receptors

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Gene delivery to the rat retina by non-viral vectors based on chloroquine-containing cationic niosomes

Mashal, M., Attia, N., Martínez-Navarrete, G., Soto-Sánchez, C., Fernández, E., Grijalvo, S., Eritja, R., Puras, G., & Pedraz, J. L. (2019) · Journal of Controlled Release · doi:10.1016/j.jconrel.2019.05.010 · Cited by 58 · see citing works

Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders

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Characterization of AAV vectors: a review of analytical techniques and critical quality attributes

Kontogiannis, T., Braybrook, J., McElroy, C., Foy, C., Whale, A. S., Quaglia, M., & Smales, C. M. (2024) · Molecular Therapy - Methods &amp; Clinical Development · doi:10.1016/j.omtm.2024.101309 · Cited by 57 · see citing works

Immune responses to gene editing by viral and non-viral delivery vectors used in retinal gene therapy

Ren, D., Fisson, S., Dalkara, D., & Ail, D. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14091973 · Cited by 52 · see citing works

Ocular drug delivery: advancements and innovations

Tian, B., Bilsbury, E., Doherty, S., Teebagy, S., Wood, E., Su, W., Gao, G., & Lin, H. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14091931 · Cited by 50 · see citing works

Update on viral gene therapy clinical trials for retinal diseases

Cheng, S.-Y., & Punzo, C. (2022) · Human Gene Therapy · doi:10.1089/hum.2022.159 · Cited by 49 · see citing works

Innate immune sensing of adeno-associated virus vectors

Cao, D., Byrne, B. J., de Jong, Y. P., Terhorst, C., Duan, D., Herzog, R. W., & Kumar, S. R. P. (2024) · Human Gene Therapy · doi:10.1089/hum.2024.040 · Cited by 50 · see citing works

Retinal disease in ciliopathies: Recent advances with a focus on stem cell-based therapies

Chen, H. Y., Welby, E., Li, T., & Swaroop, A. (2019) · Translational Science of Rare Diseases · doi:10.3233/trd-190038 · Cited by 44 · see citing works

Advances in ophthalmic optogenetics: approaches and applications

Prosseda, P. P., Tran, M., Kowal, T., Wang, B., & Sun, Y. (2022) · Biomolecules · doi:10.3390/biom12020269 · Cited by 41 · see citing works

A qPCR method for AAV genome titer with ddPCR-level of accuracy and precision

Wang, Y., Menon, N., Shen, S., Feschenko, M., & Bergelson, S. (2020) · Molecular Therapy - Methods &amp; Clinical Development · doi:10.1016/j.omtm.2020.09.017 · Cited by 34 · see citing works

An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

Sun, J., Shao, W., Chen, X., Merricks, E. P., Wimsey, L., Abajas, Y. L., Niemeyer, G. P., Lothrop, C. D., Monahan, P. E., Samulski, R. J., et al. (2018) · Molecular Therapy - Methods &amp; Clinical Development · doi:10.1016/j.omtm.2018.07.011 · Cited by 30 · see citing works

Gene Therapy for Inherited Retinal Degeneration

Arbabi, A., Liu, A., & Ameri, H. (2019) · Journal of Ocular Pharmacology and Therapeutics · doi:10.1089/jop.2018.0087 · Cited by 30 · see citing works

Sex Difference Leads to Differential Gene Expression Patterns and Therapeutic Efficacy in Mucopolysaccharidosis IVA Murine Model Receiving AAV8 Gene Therapy

Piechnik, M., Amendum, P. C., Sawamoto, K., Stapleton, M., Khan, S., Fnu, N., Álvarez, V., Pachon, A. M. H., Danos, O., Bruder, J. T., et al. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms232012693 · Cited by 28 · see citing works

Optimization of the quality by design approach for gene therapy products: A case study for adeno-associated viral vectors

Tanaka, T., Hanaoka, H., & Sakurai, S. (2020) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2020.08.002 · Cited by 27 · see citing works

Holographic Imaging of Insect Cell Cultures: Online Non-Invasive Monitoring of Adeno-Associated Virus Production and Cell Concentration

Pais, D. A. M., Galrão, P. R. S., Kryzhanska, A., Barbau, J., Isidro, I. A., & Alves, P. M. (2020) · Processes · doi:10.3390/pr8040487 · Cited by 25 · see citing works

Viral-mediated gene therapy and genetically modified therapeutics: A primer on biosafety handling for the health-system pharmacist

Blind, J. E., McLeod, E. N., & Campbell, K. J. (2019) · American Journal of Health-System Pharmacy · doi:10.1093/ajhp/zxz056 · Cited by 26 · see citing works

Modulation of the liver immune microenvironment by the adeno-associated virus serotype 8 gene therapy vector

Carestia, A., Kim, S.-J., Horling, F., Rottensteiner, H., Lubich, C., Reipert, B. M., Crowe, B. A., & Jenne, C. N. (2021) · Molecular Therapy - Methods &amp; Clinical Development · doi:10.1016/j.omtm.2020.10.023 · Cited by 24 · see citing works

AAV2 and AAV9 tropism and transgene expression in the mouse eye and major tissues after intravitreal and subretinal delivery

Koponen, S., Kokki, E., Tamminen, T., & Ylä-Herttuala, S. (2023) · Frontiers in Drug Delivery · doi:10.3389/fddev.2023.1148795 · Cited by 21 · see citing works

Retina transduction by rAAV2 after intravitreal injection: Comparison between mouse and rat

Dias, M. S., Araujo, V. G., Vasconcelos, T., Li, Q., Hauswirth, W. W., Linden, R., & Petrs-Silva, H. (2019) · Gene Therapy · doi:10.1038/s41434-019-0100-9 · Cited by 17 · see citing works

Engineered Extracellular Vesicles/Exosomes as a New Tool against Neurodegenerative Diseases

Ferrantelli, F., Chiozzini, C., Leone, P., Manfredi, F., & Federico, M. (2020) · Pharmaceutics · doi:10.3390/pharmaceutics12060529 · Cited by 17 · see citing works

Neutralisation of adeno-associated virus transduction by human vitreous humour

Andrzejewski, S., Moyle, P. M., Stringer, B. W., Steel, J. C., & Layton, C. J. (2021) · Gene Therapy · doi:10.1038/s41434-020-0162-8 · Cited by 10 · see citing works

Prevention and inhibition of adverse humoral immune response to gene therapy mediated by adeno-associated virus (AAV) vector

Meliani, A. (2026) · doi:10.70675/83ee599czfe90z44e6za201z0938409e1748

Gene therapy for cardiovascular applications

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Clinical gene therapy using recombinant adeno-associated virus vectors

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The Influence Of Adeno Associated Virus Vector Receptor AAVR And Co-Receptors And Their Mutations On Tropism And Transduction Of Adeno Associated Virus During Gene Therapy A Review Tirthal Rai Usha Adiga Deepika Kamath M

Tirthal, U. A. (2022) · Journal of Pharmaceutical Negative Results · doi:10.47750/pnr.2022.13.s08.489

A Comparison of Inducible Gene Expression Platforms: Implications for Recombinant Adeno-Associated Virus (rAAV) Vector-Mediated Ocular Gene Therapy

Lipinski, D. M. (2019) · Advances in Experimental Medicine and Biology · doi:10.1007/978-3-030-27378-1_13 · Cited by 8 · see citing works

Onco-Retroviral and Lentiviral Vector-Based Gene Therapy for Hemophilia: Preclinical Studies

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