Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles

Improved methods for building protein models in electron density maps and the location of errors in these models

Jones, T. A., Zou, J.-Y., Cowan, S. W., & Kjeldgaard, M. (1991) · Acta Crystallographica Section A Foundations of Crystallography · doi:10.1107/s0108767390010224 · Cited by 12,650 · see citing works

SWISS-MODEL: an automated protein homology-modeling server

Schwede, T. F. (2003) · Nucleic Acids Research · doi:10.1093/nar/gkg520 · Cited by 5,592 · see citing works

Rapid evolution of a protein in vitro by DNA shuffling

Stemmer, W. P. C. (1994) · Nature · doi:10.1038/370389a0 · Cited by 1,932 · 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

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 · see citing works

Improved Green Fluorescent Protein by Molecular Evolution Using DNA Shuffling

Crameri, A., Whitehorn, E. A., Tate, E. H., & Stemmer, W. P. C. (1996) · Nature Biotechnology · doi:10.1038/nbt0396-315 · Cited by 1,187 · 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

DNA shuffling of a family of genes from diverse species accelerates directed evolution

Crameri, A., Raillard, S. A., Bermudez, E., & Stemmer, W. P. C. (1998) · Nature · doi:10.1038/34663 · Cited by 826 · see citing works

Isolation of adenovirus type 5 host range deletion mutants defective for transformation of rat embryo cells

Jones, N. C., & Shenk, T. E. (1979) · Cell · doi:10.1016/0092-8674(79)90275-7 · Cited by 784 · see citing works

Unraveling hot spots in binding interfaces: progress and challenges

DeLano, W. L. (2002) · Current Opinion in Structural Biology · doi:10.1016/s0959-440x(02)00283-x · Cited by 767 · see citing works

Cross-Packaging of a Single Adeno-Associated Virus (AAV) Type 2 Vector Genome into Multiple AAV Serotypes Enables Transduction with Broad Specificity

Rabinowitz, J. E., Rolling, F., Li, C., Conrath, H., Xiao, W., Xiao, X., & Samulski, R. J. (2002) · Journal of Virology · doi:10.1128/jvi.76.2.791-801.2002 · Cited by 762 · 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

αVβ5 integrin: a co-receptor for adeno-associated virus type 2 infection

Summerford, C., Bartlett, J. S., & Samulski, R. J. (1999) · Nature Medicine · doi:10.1038/4768 · Cited by 711 · 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

Directed evolution of adeno-associated virus yields enhanced gene delivery vectors

Maheshri, N., Koerber, J. T., Kaspar, B. K., & Schaffer, D. V. (2006) · Nature Biotechnology · doi:10.1038/nbt1182 · Cited by 538 · 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

New recombinant serotypes of AAV vectors

Gao, G., Vandenberghe, L., & Wilson, J. (2005) · Current Gene Therapy · doi:10.2174/1566523054065057 · Cited by 487 · see citing works

Two Distinct Subpopulations of Nestin-Positive Cells in Adult Mouse Dentate Gyrus

Fukuda, S., Kato, F., Tozuka, Y., Yamaguchi, M., Miyamoto, Y., & Hisatsune, T. (2003) · Journal of Neuroscience · doi:10.1523/jneurosci.23-28-09357.2003 · Cited by 423 · see citing works

Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors

Müller, O. J., Kaul, F., Weitzman, M. D., Pasqualini, R., Arap, W., Kleinschmidt, J. A., & Trepel, M. (2003) · Nature Biotechnology · doi:10.1038/nbt856 · Cited by 411 · see citing works

Cooperative effects of INK4a and ras in melanoma susceptibility in vivoPreprint

Chin, L., Pomerantz, J. H., Polsky, D., Jacobson, M., Cohen, C., Cordon‐Cardo, C., Horner, J. W., & DePinho, R. A. (1997) · Genes & Development · doi:10.1101/gad.11.21.2822 · Cited by 403 · see citing works

Identification of a heparin-binding motif on adeno-associated virus type 2 capsids

Kern, A., Schmidt, K., Leder, C., Müller, O. J., Wobus, C. E., Bettinger, K., Von der Lieth, C. W., King, J. A., & Kleinschmidt, J. A. (2003) · Journal of Virology · doi:10.1128/jvi.77.20.11072-11081.2003 · Cited by 380 · see citing works

Identification of amino acid residues in the capsid proteins of adeno-associated virus type 2 that contribute to heparan sulfate proteoglycan binding

Opie, S. R., Warrington, Agbandje-McKenna, M., Zolotukhin, S., & Muzyczka, N. (2003) · Journal of Virology · doi:10.1128/jvi.77.12.6995-7006.2003 · Cited by 364 · see citing works

Biology of Adeno-associated Virus

Berns, K. I., & Giraud, C. (1996) · Current topics in microbiology and immunology · doi:10.1007/978-3-642-80207-2_1 · Cited by 308 · see citing works

Monoclonal Antibodies against the Adeno-Associated Virus Type 2 (AAV-2) Capsid: Epitope Mapping and Identification of Capsid Domains Involved in AAV-2–Cell Interaction and Neutralization of AAV-2 Infection

Wobus, C. E., Hügle-Dörr, B., Girod, A., Petersen, G., Hallek, M., & Kleinschmidt, J. A. (2000) · Journal of Virology · doi:10.1128/jvi.74.19.9281-9293.2000 · Cited by 273 · see citing works

Role of α v Integrins in Adenovirus Cell Entry and Gene Delivery

Nemerow, G. R., & Stewart, P. L. (1999) · Microbiology and Molecular Biology Reviews · doi:10.1128/mmbr.63.3.725-734.1999 · Cited by 270 · 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

In vitro selection of viral vectors with modified tropism: the adeno-associated virus display

Perabò, L., Büning, H., Kofler, D. M., Ried, M. U., Girod, A., Wendtner, C., Enssle, J., & Hallek, M. J. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(03)00123-0 · Cited by 232 · see citing works

Nestin expression in reactive astrocytes following focal cerebral ischemia in rats

Duggal, N., Schmidt‐Kastner, R., & Hakim, A. M. (1997) · Brain Research · doi:10.1016/s0006-8993(97)00588-x · Cited by 208 · 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

Structurally mapping the diverse phenotype of adeno-associated virus serotype 4

Govindasamy, L., Padron, E., McKenna, R., Muzyczka, N., Kaludov, N., Chiorini, J. A., & Agbandje-McKenna, M. (2006) · Journal of Virology · doi:10.1128/jvi.01536-06 · Cited by 205 · see citing works

Mutations on the External Surfaces of Adeno-AssociatedVirus Type 2 Capsids That Affect Transduction andNeutralization

Lochrie, M. A., Tatsuno, G. P., Christie, B., McDonnell, J. W., Zhou, S., Surosky, R., Pierce, G. F., & Colosi, P. (2006) · Journal of Virology · doi:10.1128/jvi.80.2.821-834.2006 · Cited by 201 · see citing works

Efficient Neuronal Gene Transfer with AAV8 Leads to Neurotoxic Levels of Tau or Green Fluorescent Proteins

Klein, R. L., Dayton, R. D., Leidenheimer, N. J., Jansen, K., Golde, T. E., & Zweig, R. M. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2005.10.008 · Cited by 200 · see citing works

AAV hybrid serotypes: improved vectors for gene delivery

Choi, V., McCarty, D., & Samulski, R. (2005) · Current Gene Therapy · doi:10.2174/1566523054064968 · Cited by 191 · see citing works

Cross-Dressing the Virion: the Transcapsidation of Adeno-Associated Virus Serotypes Functionally Defines Subgroups

Rabinowitz, J. E., Bowles, D. E., Faust, S. M., Ledford, J. G., Cunningham, S. E., & Samulski, R. J. (2004) · Journal of Virology · doi:10.1128/jvi.78.9.4421-4432.2004 · Cited by 172 · see citing works

Cranial irradiation after surgical excision of brain metastases in melanoma patients

Skibber, J. M., Soong, S.-J., Austin, L., Balch, C. M., & Sawaya, R. A. (1996) · Annals of Surgical Oncology · doi:10.1007/bf02305789 · Cited by 149 · 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

Insertional Mutagenesis of AAV2 Capsid and the Production of Recombinant Virus

Rabinowitz, J. E., Xiao, W., & Samulski, R. J. (1999) · Virology · doi:10.1006/viro.1999.0045 · Cited by 136 · see citing works

Combinatorial engineering of a gene therapy vector: directed evolution of adeno‐associated virus

Perabo, L., Endell, J., King, S., Lux, K., Goldnau, D., Hallek, M., & Büning, H. (2006) · The Journal of Gene Medicine · doi:10.1002/jgm.849 · Cited by 136 · see citing works

Conjugate-Based Targeting of Recombinant Adeno-Associated Virus Type 2 Vectors by Using Avidin-Linked Ligands

Ponnazhagan, S., Mahendra, G., Kumar, S., Thompson, J. A., & Castillas, M. (2002) · Journal of Virology · doi:10.1128/jvi.76.24.12900-12907.2002 · Cited by 129 · see citing works

Analysis of shuffled gene libraries

Joern, J. M., Meinhold, P., & Arnold, F. H. (2002) · Journal of Molecular Biology · doi:10.1006/jmbi.2001.5349 · Cited by 101 · see citing works

Construction and evolution of antibody–phage libraries by DMA shuffling

Crameri, A., Cwirla, S., & Stemmer, W. P. C. (1996) · Nature Medicine · doi:10.1038/nm0196-100 · Cited by 98 · see citing works

Both products of the mouse Ink4a/Arf locus suppress melanoma formation in vivo

Sharpless, N. E., Kannan, K., Xu, J., Bosenberg, M., & Chin, L. (2003) · Oncogene · doi:10.1038/sj.onc.1206809 · Cited by 97 · see citing works

Lack of Extracellular Signal-Regulated Kinase Mitogen-Activated Protein Kinase Signaling Shows a New Type of Melanoma

Shields, J. M., Thomas, N. E., Cregger, M. A., Berger, A. J., Leslie, M. C., Torrice, C., Hao, H., Penland, S., Arbiser, J. L., Scott, G., et al. (2007) · Cancer Research · doi:10.1158/0008-5472.can-06-3311 · Cited by 81 · see citing works

Marker Rescue of Adeno-Associated Virus (AAV) Capsid Mutants: a Novel Approach for Chimeric AAV Production

Bowles, D. E., Rabinowitz, J. E., & Samulski, R. J. (2002) · Journal of Virology · doi:10.1128/jvi.77.1.423-432.2003 · Cited by 67 · see citing works

Enhancing rAAV vector expression in the lung

Virella‐Lowell, I., Zusman, B. D., Foust, K. D., Loiler, S., Conlon, T. J., Song, S., Chesnut, K. A., Ferkol, T. W., & Flotte, T. R. (2005) · The Journal of Gene Medicine · doi:10.1002/jgm.759 · Cited by 62 · see citing works

Recent Developments in Recombinant AAV-Mediated Gene Therapy for Lung Diseases

Flotte, T. R. (2005) · Current Gene Therapy · doi:10.2174/1566523054064986 · Cited by 45 · see citing works

5-Bromo-2-deoxyuridine regulates invasiveness and expression of integrins and matrix-degrading proteinases in a differentiated hamster melanoma cell

Thomas, L. A., Chan, P. W., Chang, S., & Damsky, C. H. (1993) · Journal of Cell Science · doi:10.1242/jcs.105.1.191 · Cited by 38 · see citing works

Current Development of Adeno-Associated Viral Vectors

Romano, G. (2005) · Drug News & Perspectives · doi:10.1358/dnp.2005.18.5.917326 · Cited by 23 · see citing works

AAV Vector Delivery to Cells in Culture

Smith, A. D., Collaco, R. F., & Trempe, J. P. (2004) · Humana Press eBooks · doi:10.1385/1-59259-650-9:167 · Cited by 12 · see citing works

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

Foust, K. D., Nurre, E., Montgomery, C. L., Hernandez, A., Chan, C. M., & Kaspar, B. K. (2009) · Nature Biotechnology · doi:10.1038/nbt.1515 · Cited by 1,456 · 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

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

State-of-the-art gene-based therapies: the road ahead

Kay, M. A. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2971 · Cited by 672 · 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

Therapeutic in vivo delivery of gene editing agents

Raguram, A., Banskota, S., & Liu, D. R. (2022) · Cell · doi:10.1016/j.cell.2022.03.045 · Cited by 532 · see citing works

Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates

Gray, S. J., Matagne, V., Bachaboina, L., Yadav, S., Ojeda, S. R., & Samulski, R. J. (2011) · Molecular Therapy · doi:10.1038/mt.2011.72 · Cited by 501 · see citing works

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

Lisowski, L., Dane, A. P., Chu, K., Zhang, Y., Cunningham, S. C., Wilson, E. M., Nygaard, S., Grompe, M., Alexander, I. E., & Kay, M. A. (2014) · Nature · doi:10.1038/nature12875 · Cited by 450 · see citing works

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

Bowles, D. E., McPhee, S. W., Li, C., Gray, S. J., Samulski, J. J., Camp, A. S., Li, J., Wang, B., Monahan, P. E., Rabinowitz, J. E., et al. (2012) · Molecular Therapy · doi:10.1038/mt.2011.237 · Cited by 400 · see citing works

AAV vectors: The Rubik’s cube of human gene therapy

Pupo, A., Fernández, A., Low, S. H., François, A., Suárez-Amarán, L., & Samulski, R. J. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.09.015 · Cited by 361 · see citing works

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

Keeler, A. M., & Flotte, T. R. (2019) · Annual Review of Virology · doi:10.1146/annurev-virology-092818-015530 · Cited by 322 · see citing works

In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector

Zinn, E., Pacouret, S., Khaychuk, V., Turunen, H. T., Carvalho, L. S., Andres-Mateos, E., Shah, S., Shelke, R., Maurer, A. C., Plovie, E., et al. (2015) · Cell Reports · doi:10.1016/j.celrep.2015.07.019 · Cited by 310 · see citing works

Gene therapy for neurological disorders: progress and prospects

Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M., & Sah, D. W. Y. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.110 · Cited by 306 · see citing works

Preferential labeling of inhibitory and excitatory cortical neurons by endogenous tropism of adeno-associated virus and lentivirus vectors

Nathanson, J. L., Yanagawa, Y., Obata, K., & Callaway, E. M. (2009) · Neuroscience · doi:10.1016/j.neuroscience.2009.03.032 · Cited by 305 · 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

A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype

Ellis, B. L., Hirsch, M. L., Barker, J. C., Connelly, J. P., Steininger, R. J., & Porteus, M. H. (2013) · Virology Journal · doi:10.1186/1743-422x-10-74 · Cited by 252 · see citing works

Immune responses to retinal gene therapy using adeno-associated viral vectors – implications for treatment success and safety

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

Erratum: Gene therapy for neurological disorders: progress and prospects

Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M., & Sah, D. W. Y. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.158 · Cited by 248 · see citing works

Adeno-associated virus (AAV) vectors in cancer gene therapy

Santiago-Ortiz, J. L., & Schaffer, D. V. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.01.001 · Cited by 231 · 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

Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia

Li, C., Narkbunnam, N., Samulski, R. J., Asokan, A., Hu, G., Jacobson, L. J., Manco-Johnson, M. J., Monahan, P. E., & The Joint Outcome Study Investigators (2012) · Gene Therapy · doi:10.1038/gt.2011.90 · Cited by 225 · see citing works

Recombination in Eukaryotic Single Stranded DNA Viruses

Martin, D. P., Biagini, P., Lefeuvre, P., Golden, M., Roumagnac, P., & Varsani, A. (2011) · Viruses · doi:10.3390/v3091699 · Cited by 225 · see citing works

A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection

Yang, L., Jiang, J., Drouin, L. M., Agbandje‐McKenna, M., Chen, C., Qiao, C., Pu, D., Hu, X., Wang, D.-Z., Li, J., et al. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0813207106 · Cited by 208 · see citing works

An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery

Huang, Q., Chan, K. Y., Wu, J. H., Botticello-Romero, N. R., Zheng, Q., Lou, S., Keyes, C., Svanbergsson, A., Johnston, J., Mills, A., et al. (2024) · Science · doi:10.1126/science.adm8386 · Cited by 200 · see citing works

Sarcoplasmic reticulum Ca2+ATPase as a therapeutic target for heart failure

Lipskaia, L., Chemaly, E. R., Hadri, L., Lompré, A., & Hajjar, R. J. (2009) · Expert Opinion on Biological Therapy · doi:10.1517/14712590903321462 · Cited by 193 · see citing works

Molecular evolution of adeno-associated virus for enhanced glial gene delivery

Koerber, J. T., Klimczak, R., Jang, J.-H., Dalkara, D., Flannery, J. G., & Schaffer, D. V. (2009) · Molecular Therapy · doi:10.1038/mt.2009.184 · Cited by 181 · see citing works

Directed Evolution of a Novel Adeno-associated Virus (AAV) Vector That Crosses the Seizure-compromised Blood–Brain Barrier (BBB)

Gray, S. J., Blake, B. L., Criswell, H. E., Nicolson, S. C., Samulski, R. J., & McCown, T. J. (2010) · Molecular Therapy · doi:10.1038/mt.2009.292 · Cited by 185 · see citing works

DNA Shuffling of Adeno-associated Virus Yields Functionally Diverse Viral Progeny

Koerber, J. T., Jang, J.-H., & Schaffer, D. V. (2008) · Molecular Therapy · doi:10.1038/mt.2008.167 · Cited by 181 · 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

Directed evolution of adeno-associated virus to an infectious respiratory virus

Excoffon, K., Koerber, J. T., Dickey, D. D., Murtha, M., Keshavjee, S., Kaspar, B., Zabner, J., & Schaffer, D. (2009) · Proceedings of the National Academy of Sciences of the United States of America · doi:10.1073/pnas.0813365106 · Cited by 190 · see citing works

Protein Cages: From Fundamentals to Advanced Applications

Edwardson, T. G. W., Levasseur, M. D., Tetter, S., Steinauer, A., Hori, M., & Hilvert, D. (2022) · Chemical Reviews · doi:10.1021/acs.chemrev.1c00877 · Cited by 169 · see citing works

Tailoring the AAV vector capsid for gene therapy

Vandenberghe, L. H., Wilson, J. M., & Gao, G. (2009) · Gene Therapy · doi:10.1038/gt.2008.170 · Cited by 155 · 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

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity

Paulk, N. K., Pekrun, K., Zhu, E., Nygaard, S., Li, B., Xu, J., Chu, K., Leborgne, C., Dane, A. P., Haft, A., et al. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2017.09.021 · Cited by 161 · see citing works

Efficient recovery of dysferlin deficiency by dual adeno-associated vector-mediated gene transfer

Lostal, W., Bartoli, M., Bourg, N., Roudaut, C., Bentaïb, A., Miyake, K., Guerchet, N., Fougerousse, F., McNeil, P., & Richard, I. (2010) · Human Molecular Genetics · doi:10.1093/hmg/ddq065 · Cited by 173 · 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 · see citing works

Adeno-Associated Virus Toolkit to Target Diverse Brain Cells

Challis, R. C., Ravindra Kumar, S., Chen, X., Goertsen, D., Coughlin, G. M., Hori, A. M., Chuapoco, M. R., Otis, T. S., Miles, T. F., & Gradinaru, V. (2022) · Annual Review of Neuroscience · doi:10.1146/annurev-neuro-111020-100834 · Cited by 148 · see citing works

Novel random peptide libraries displayed on AAV serotype 9 for selection of endothelial cell-directed gene transfer vectors

Varadi, K., Michelfelder, S., Korff, T., Hecker, M., Trepel, M., Katus, H. A., Kleinschmidt, J. A., & Müller, O. J. (2011) · Gene Therapy · doi:10.1038/gt.2011.143 · Cited by 145 · see citing works

Recent progress and considerations for AAV gene therapies targeting the central nervous system

Lykken, E. A., Shyng, C., Edwards, R. J., Rozenberg, A. J., & Gray, S. J. (2018) · Journal of Neurodevelopmental Disorders · doi:10.1186/s11689-018-9234-0 · Cited by 140 · see citing works

A systematic capsid evolution approach performed in vivo for the design of AAV vectors with tailored properties and tropism

Davidsson, M., Wang, G., Aldrin-Kirk, P., Cardoso, T., Nolbrant, S., Hartnor, M., Mudannayake, J., Parmar, M., & Björklund, T. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1910061116 · Cited by 142 · 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 (AAV) Versus Immune Response

Rabinowitz, J., Chan, Y. K., & Samulski, R. J. (2019) · Viruses · doi:10.3390/v11020102 · Cited by 122 · see citing works

Adeno-associated virus: fit to serve

Zinn, E. M., & Vandenberghe, L. H. (2014) · Current Opinion in Virology · doi:10.1016/j.coviro.2014.07.008 · Cited by 122 · see citing works

Gene Therapy: Design and Prospects for Craniofacial Regeneration

Scheller, E. L., & Krebsbach, P. H. (2009) · Journal of Dental Research · doi:10.1177/0022034509337480 · Cited by 119 · see citing works

Engineering Delivery Vehicles for Genome Editing

Nelson, C. E., & Gersbach, C. A. (2016) · Annual Review of Chemical and Biomolecular Engineering · doi:10.1146/annurev-chembioeng-080615-034711 · Cited by 118 · see citing works

Adeno-Associated Virus Structural Biology as a Tool in Vector Development

Drouin, L. M., & Agbandje-McKenna, M. (2013) · Future Virology · doi:10.2217/fvl.13.112 · Cited by 114 · see citing works

In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy

Choudhury, S., Fitzpatrick, Z., Harris, A. F., Maitland, S. A., Ferreira, J. S., Zhang, Y., Ma, S., Sharma, R. B., Gray‐Edwards, H. L., Johnson, J. A., et al. (2016) · Molecular Therapy · doi:10.1038/mt.2016.84 · Cited by 115 · see citing works

Engineering the AAV capsid to optimize vector–host-interactions

Büning, H., Huber, A., Zhang, L. N., Meumann, N., & Hacker, U. T. (2015) · Current Opinion in Pharmacology · doi:10.1016/j.coph.2015.08.002 · Cited by 113 · see citing works

E Pluribus Unum: 50 Years of Research, Millions of Viruses, and One Goal—Tailored Acceleration of AAV Evolution

Grimm, D., & Zolotukhin, S. (2015) · Molecular Therapy · doi:10.1038/mt.2015.173 · Cited by 107 · see citing works

Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids

Wang, L., Bell, P. L., Somanathan, S., Wang, Q., He, Z., Yu, H., McMenamin, D., Goode, T. L., Calcedo, R., & Wilson, J. M. (2015) · Molecular Therapy · doi:10.1038/mt.2015.179 · Cited by 107 · see citing works

A Precise Chemical Strategy To Alter the Receptor Specificity of the Adeno‐Associated Virus

Kelemen, R. E., Mukherjee, R., Cao, X., Erickson, S. B., Zheng, Y., & Chatterjee, A. (2016) · Angewandte Chemie International Edition · doi:10.1002/anie.201604067 · Cited by 97 · see citing works

Viral Vectors for Gene Transfer: Current Status of Gene Therapeutics

Heilbronn, R., & Weger, S. (2009) · Handbook of experimental pharmacology · doi:10.1007/978-3-642-00477-3_5 · Cited by 98 · see citing works

RNA Encapsidation by SV40-Derived Nanoparticles Follows a Rapid Two-State Mechanism

Kler, S., Asor, R., Li, C., Ginsburg, A., Harries, D., Oppenheim, A., Zlotnick, A., & Raviv, U. (2012) · Journal of the American Chemical Society · doi:10.1021/ja2110703 · Cited by 98 · see citing works

Structural insights into adeno-associated virus serotype 5

Govindasamy, L., DiMattia, M. A., Gurda, B. L., Halder, S., McKenna, R., Chiorini, J. A., Muzyczka, N., Zolotukhin, S., & Agbandje-McKenna, M. (2013) · Journal of Virology · doi:10.1128/jvi.00867-13 · Cited by 95 · 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

Single amino acid modification of adeno-associated virus capsid changes transduction and humoral immune profiles

Li, C., Diprimio, N., Bowles, D. E., Hirsch, M. L., Monahan, P. E., Asokan, A., Rabinowitz, J., Agbandje-McKenna, M., & Samulski, R. J. (2012) · Journal of Virology · doi:10.1128/jvi.00675-12 · Cited by 96 · see citing works

Gene Therapy for the Nervous System: Challenges and New Strategies

Maguire, C. A., Ramirez, S. H., Merkel, S. F., Sena‐Esteves, M., & Breakefield, X. O. (2014) · Neurotherapeutics · doi:10.1007/s13311-014-0299-5 · Cited by 96 · see citing works

AAVs Anatomy: Roadmap for Optimizing Vectors for Translational Success

Mitchell, A. M., Nicolson, S. C., Warischalk, J. K., & Samulski, R. J. (2010) · Current Gene Therapy · doi:10.2174/156652310793180706 · Cited by 96 · see citing works

Using a barcoded AAV capsid library to select for clinically relevant gene therapy vectors

Pekrun, K., de Alencastro, G., Luo, Q.-J., Liu, J., Kim, Y., Nygaard, S. C., Galivo, F., Zhang, F., Song, R., Tiffany, M. R., et al. (2019) · JCI Insight · doi:10.1172/jci.insight.131610 · Cited by 97 · see citing works

AAV9: a potential blood-brain barrier buster

Manfredsson, F. P., Rising, A. C., & Mandel, R. J. (2009) · Molecular Therapy · doi:10.1038/mt.2009.15 · Cited by 94 · see citing works

Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism

Powell, S. K., Khan, N., Parker, C. L., Samulski, R. J., Matsushima, G., Gray, S. J., & McCown, T. J. (2016) · Gene Therapy · doi:10.1038/gt.2016.62 · Cited by 93 · see citing works

Structure of AAV-DJ, a Retargeted Gene Therapy Vector: Cryo-Electron Microscopy at 4.5 Å Resolution

Lerch, T. F., O’Donnell, J., Meyer, N., Xie, Q., Taylor, K. A., Stagg, S. M., & Chapman, M. S. (2012) · Structure · doi:10.1016/j.str.2012.05.004 · Cited by 89 · see citing works

Vaccination with Human Pluripotent Stem Cells Generates a Broad Spectrum of Immunological and Clinical Responses Against Colon Cancer

Li, Y., Zeng, H., Xu, R., Liu, B., & Li, Z. (2009) · Stem Cells · doi:10.1002/stem.234 · Cited by 88 · see citing works

Generation of Novel AAV Variants by Directed Evolution for Improved CFTR Delivery to Human Ciliated Airway Epithelium

Li, W.-P., Zhang, L., Johnson, J. S., Wu, Z.-J., Grieger, J., Xiao, P.-J., Drouin, L., Agbandje-McKenna, M., Pickles, R., & Samulski, R. (2009) · Molecular Therapy · doi:10.1038/mt.2009.155 · Cited by 94 · see citing works

Adeno-associated Virus as a Mammalian DNA Vector

Salganik, M., Hirsch, M. L., & Samulski, R. J. (2015) · Microbiology Spectrum · doi:10.1128/microbiolspec.mdna3-0052-2014 · Cited by 88 · see citing works

State-of-the-art human gene therapy: part I. Gene delivery technologies.

Wang, D., & Gao, G. (2014) · PubMed · Cited by 85 · see citing works

Little Vector, Big Gene Transduction: Fragmented Genome Reassembly of Adeno-associated Virus

Hirsch, M. L., Agbandje‐McKenna, M., & Samulski, R. J. (2010) · Molecular Therapy · doi:10.1038/mt.2009.280 · Cited by 86 · see citing works

CRISPR genome engineering and viral gene delivery: A case of mutual attraction

Schmidt, F., & Grimm, D. (2015) · Biotechnology Journal · doi:10.1002/biot.201400529 · Cited by 83 · see citing works

Therapeutic applications of genetic code expansion

Huang, Y., & Liu, T. (2018) · Synthetic and Systems Biotechnology · doi:10.1016/j.synbio.2018.09.003 · Cited by 82 · see citing works

Fantastic AAV Gene Therapy Vectors and How to Find Them—Random Diversification, Rational Design and Machine Learning

Becker, J., Fakhiri, J., & Grimm, D. (2022) · Pathogens · doi:10.3390/pathogens11070756 · Cited by 80 · see citing works

Ferret and Pig Models of Cystic Fibrosis: Prospects and Promise for Gene Therapy

Yan, Z.-Y., Stewart, Z., Sinn, P., Olsen, J., Hu, J., McCray, P., & Engelhardt, J. (2014) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2014.154 · Cited by 75 · see citing works

Current progress and limitations of AAV mediated delivery of protein therapeutic genes and the importance of developing quantitative pharmacokinetic/pharmacodynamic (PK/PD) models

Chowdhury, E. A., Meno‐Tetang, G. M. L., Chang, H. Y., Wu, S., Huang, H. W., Jamier, T., Chandran, J. S., & Shah, D. K. (2021) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2021.01.017 · Cited by 78 · see citing works

Successful Expansion but Not Complete Restriction of Tropism of Adeno-Associated Virus by In Vivo Biopanning of Random Virus Display Peptide Libraries

Michelfelder, S., Kohlschütter, J., Skorupa, A., Pfennings, S., Müller, O. J., Kleinschmidt, J. A., & Trepel, M. (2009) · PLoS ONE · doi:10.1371/journal.pone.0005122 · Cited by 75 · see citing works

Vector Design Tour de Force: Integrating Combinatorial and Rational Approaches to Derive Novel Adeno-associated Virus Variants

Marsic, D., Lakshmanan Govindasamy, A., Currlin, S., Markusic, D. M., Tseng, Y.-S., Herzog, R. W., Agbandje‐McKenna, M., & Zolotukhin, S. (2014) · Molecular Therapy · doi:10.1038/mt.2014.139 · Cited by 73 · see citing works

Genome engineering: a new approach to gene therapy for neuromuscular disorders

Nelson, C. E., Robinson-Hamm, J. N., & Gersbach, C. A. (2017) · Nature Reviews Neurology · doi:10.1038/nrneurol.2017.126 · Cited by 71 · see citing works

The next step in gene delivery: Molecular engineering of adeno-associated virus serotypes

Wang, J., Faust, S. M., & Rabinowitz, J. E. (2010) · Journal of Molecular and Cellular Cardiology · doi:10.1016/j.yjmcc.2010.10.017 · Cited by 69 · see citing works

Rationale and strategies for the development of safe and effective optimized AAV vectors for human gene therapy

Srivastava, A. (2023) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2023.05.014 · Cited by 65 · see citing works

Heart-targeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library

Ying, Y., Müller, O. J., Goehringer, C., Leuchs, B., Trepel, M., Katus, H. A., & Kleinschmidt, J. A. (2010) · Gene Therapy · doi:10.1038/gt.2010.44 · Cited by 69 · see citing works

Structure of neurotropic adeno-associated virus AAVrh.8

Halder, S., Vliet, K. V., Smith, J. K., Duong, T. T. P., McKENNA, R. V., Wilson, J. M., & Agbandje‐McKenna, M. (2015) · Journal of Structural Biology · doi:10.1016/j.jsb.2015.08.017 · Cited by 62 · see citing works

Adeno-Associated Virus Biology

Weitzman, M. D., & Linden, R. M. (2012) · Methods in Molecular Biology · doi:10.1007/978-1-61779-370-7_1 · Cited by 132 · see citing works

Engineered antibody therapies to counteract mutant huntingtin and related toxic intracellular proteins

Butler, D. C., McLear, J. A., & Messer, A. C. (2011) · Progress in Neurobiology · doi:10.1016/j.pneurobio.2011.11.004 · Cited by 62 · see citing works

Regulatory and Scientific Advancements in Gene Therapy: State-of-the-Art of Clinical Applications and of the Supporting European Regulatory Framework

Carvalho, M. A. B. R., Sepodes, B. M. N., & Martins, A. P. (2017) · Frontiers in Medicine · doi:10.3389/fmed.2017.00182 · Cited by 64 · see citing works

Viral vectors as vaccine carriers

Ertl, H. C. J. (2016) · Current Opinion in Virology · doi:10.1016/j.coviro.2016.06.001 · Cited by 59 · see citing works

Relevance of Assembly-Activating Protein for Adeno-associated Virus Vector Production and Capsid Protein Stability in Mammalian and Insect Cells

Große, S., Penaud‐Budloo, M., Herrmann, A.-K., Börner, K., Fakhiri, J., Laketa, V., Krämer, C., Wiedtke, E., Gunkel, M., Ménard, L., et al. (2017) · Journal of Virology · doi:10.1128/jvi.01198-17 · Cited by 59 · see citing works

A versatile toolkit for overcoming AAV immunity

Li, X., Wei, X., Lin, J., & Ou, L. (2022) · Frontiers in Immunology · doi:10.3389/fimmu.2022.991832 · Cited by 57 · see citing works

Latest developments in the large-scale production of adeno-associated virus vectors in insect cells toward the treatment of neuromuscular diseases

Galibert, L., & Merten, O. (2011) · Journal of Invertebrate Pathology · doi:10.1016/j.jip.2011.05.008 · Cited by 56 · see citing works

Applying machine learning to predict viral assembly for adeno-associated virus capsid libraries

Marques, A. D., Kummer, M., Kondratov, O., Banerjee, A., Moskalenko, O. V., & Zolotukhin, S. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.11.017 · Cited by 55 · see citing works

Gene delivery methods in cardiac gene therapy

Ishikawa, K., Tilemann, L. M., Fish, K. M., & Hajjar, R. J. (2011) · The Journal of Gene Medicine · doi:10.1002/jgm.1609 · Cited by 55 · 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

Immune responses to adeno-associated virus type 2 encoding channelrhodopsin-2 in a genetically blind rat model for gene therapy

Sugano, E., Isago, H., Wang, Z., Murayama, N., Tamai, M., & Tomita, H. (2010) · Gene Therapy · doi:10.1038/gt.2010.140 · Cited by 55 · see citing works

Synthetic virology: engineering viruses for gene delivery

Guenther, C. M., Kuypers, B. E., Lam, M. T., Robinson, T. M., Zhao, J. X., & Suh, J. (2014) · Wiley Interdisciplinary Reviews Nanomedicine and Nanobiotechnology · doi:10.1002/wnan.1287 · Cited by 54 · see citing works

Next-generation AAV vectors for clinical use: an ever-accelerating race

Weinmann, J., & Grimm, D. (2017) · Virus Genes · doi:10.1007/s11262-017-1502-7 · Cited by 53 · 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

DNA shuffling of the GP3 genes of porcine reproductive and respiratory syndrome virus (PRRSV) produces a chimeric virus with an improved cross-neutralizing ability against a heterologous PRRSV strain

Zhou, L., Ni, Y.-Y., Piñeyro, P. E., Sanford, B. J., Cossaboom, C. M., Dryman, B. A., Huang, Y., Cao, D., & Meng, X. (2012) · Virology · doi:10.1016/j.virol.2012.09.005 · Cited by 52 · see citing works

Optimization of AAV vectors to target persistent viral reservoirs

Colón-Thillet, R., Jerome, K. R., & Stone, D. (2021) · Virology Journal · doi:10.1186/s12985-021-01555-7 · Cited by 50 · see citing works

Optimization of stealth adeno-associated virus vectors by randomization of immunogenic epitopes

Maersch, S., Huber, A., Büning, H., Hallek, M., & Perabo, L. (2010) · Virology · doi:10.1016/j.virol.2009.10.021 · Cited by 50 · see citing works

Perspective on Adeno-Associated Virus Capsid Modification for Duchenne Muscular Dystrophy Gene Therapy

Nance, M. E., & Duan, D. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.107 · Cited by 50 · see citing works

Random Insertion of mCherry Into VP3 Domain of Adeno-associated Virus Yields Fluorescent Capsids With no Loss of Infectivity

Judd, J., Wei, F., Nguyen, P. Q., Tartaglia, L. J., Agbandje‐McKenna, M., Silberg, J. J., & Suh, J. (2012) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2012.46 · Cited by 51 · see citing works

Directed evolution of adeno-associated virus for glioma cell transduction

Maguire, C. A., Gianni, D., Meijer, D. H., Shaket, L. A., Wakimoto, H., Rabkin, S. D., Gao, G., & Sena‐Esteves, M. (2009) · Journal of Neuro-Oncology · doi:10.1007/s11060-009-9972-7 · Cited by 50 · see citing works

CAV-2 Vector Development and Gene Transfer in the Central and Peripheral Nervous Systems

Rio, D. D., Beucher, B., Lavigne, M., Wehbi, A., Dopeso‐Reyes, I. G., Saggio, I., & Kremer, E. J. (2019) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2019.00071 · Cited by 50 · see citing works

In This Issue

(2008) · Molecular Therapy · doi:10.1038/mt.2008.139

743. Directed Evolution of Adeno-Associated Virus (AAV) by DNA Shuffling Yields Enhanced Gene Delivery Vectors

Li, W., & Samulski, J. R. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.08.825 · Cited by 2 · see citing works

Dissection and optimization of Adeno-associated virus (AAV) DNA family shuffling technology: The journey is the reward

Herrmann, A.-K. (2018) · heiDOK (Heidelberg University) · doi:10.11588/heidok.00025503

Secrets to finding the ideal mate:New insights into parameters that govern successful Adeno-associated virus (AAV) vector evolution

Kienle, E. (2014) · heiDOK (Heidelberg University) · doi:10.11588/heidok.00017851 · Cited by 1 · see citing works

Engineering and Evolution of Synthetic Adeno-Associated Virus (AAV) Gene Therapy Vectors via DNA Family Shuffling

Kienle, E., Senís, E., Börner, K., Niopek, D., Wiedtke, E., Große, S., & Grimm, D. (2012) · Journal of Visualized Experiments · doi:10.3791/3819 · Cited by 32 · see citing works