Cited by 225Virus-based gene therapy researchRNA Interference and Gene DeliveryImmunotherapy and Immune Responses

The DOI matches its Crossref record; 186 of the 186 references it cites were found in a registry or an index.

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

Balakrishnan, Balaji, Jayandharan, Giridhara·2014·Current Gene Therapy

DOI 10.2174/1566523214666140302193709

Open this paper in CoChat — free account

Abstract

Adeno-associated virus (AAV) based vectors have emerged as important tools for gene therapy in humans. The recent successes seen in Phase I/II clinical trials have also highlighted the issues related to the host and vector-related immune response that preclude the universal application of this promising vector system. A fundamental insight into the biological mechanisms by which AAV infects the host cell and a thorough understanding of the immediate and long-lived cellular responses to AAV infection is likely to offer clues and help design better intervention strategies to improve the therapeutic efficiency of AAV vectors. This article reviews the biology of AAV-host cellular interactions and outlines their application in the development of novel and improved AAV vector systems.

Integrity signals

Registry

Crossref · 10.2174/1566523214666140302193709

Cites

186 of 186 references verified0 citation errors · 0 not checkable

Statistics

Recomputing p-values needs the paper’s full text.

Claims the paper makes

This paper’s full text isn’t openly available, so its claims and self-check weren’t run. The registry and citation checks still apply.

Claims other papers make about this one

Each is checked against this paper’s own text: does it say what the citing paper says it does?

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine · doi:10.1038/nm1358 · Cited by 2,163
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
Effect of gene therapy on visual function in Leber’s congenital amaurosis
Bainbridge, J. W. B., Smith, A. J., Barker, S. S., Robbie, S., Henderson, R., Balaggan, K., Viswanathan, A., Holder, G. E., Stockman, A., Tyler, N., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802268 · Cited by 1,924
Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
Nathwani, A. C., Tuddenham, E. G. D., Rangarajan, S., Rosales, C., McIntosh, J., Linch, D. C., Chowdary, P., Riddell, A., Pie, A. J., Harrington, C., et al. (2011) · New England Journal of Medicine · doi:10.1056/nejmoa1108046 · Cited by 1,864
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
Gao, G.-P., Alvira, M. R., Wang, L., Calcedo, R., Johnston, J., & Wilson, J. M. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.182412299 · Cited by 1,520
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
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 · cited 1×
“Based on in vivo studies, vectors specific to retina (AAV2, AAV4 and AAV5) [64-68], brain (AAV2, AAV5, AAV8, AAV9 and AAVrh10) [69-78], cardiac tissue (AAV1, AAV2, AAV6, AAV8 and AAV9) [79-83], liver (AAV1, AAV2, AAV5, AAV6, AAV7, AAV8, AAV9 and AAV10) [49, 84-93] and lungs (AAV1, AAV2, AAV5 and AAV9) [86-89, 92-98] h…”
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
Gene therapy restores vision in a canine model of childhood blindness
Acland, G. M., Aguirre, G. D., Ray, J., Zhang, Q., Aleman, T. S., Cideciyan, A. V., Pearce-Kelling, S. E., Anand, V., Zeng, Y., Maguire, A. M., et al. (2001) · Nature Genetics · doi:10.1038/ng0501-92 · Cited by 1,202 · cited 2×
“Based on in vivo studies, vectors specific to retina (AAV2, AAV4 and AAV5) [64-68], brain (AAV2, AAV5, AAV8, AAV9 and AAVrh10) [69-78], cardiac tissue (AAV1, AAV2, AAV6, AAV8 and AAV9) [79-83], liver (AAV1, AAV2, AAV5, AAV6, AAV7, AAV8, AAV9 and AAV10) [49, 84-93] and lungs (AAV1, AAV2, AAV5 and AAV9) [86-89, 92-98] h…”
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
Transcriptional repression by YY1, a human GLI-Krüippel-related protein, and relief of repression by adenovirus E1A protein
Shi, Y., Seto, E., Chang, L., & Shenk, T. E. (1991) · Cell · doi:10.1016/0092-8674(91)90189-6 · Cited by 1,035
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 1,029
Adenovirus-Associated Defective Virus Particles
Atchison, R. W., Casto, B. C., & Hammon, W. M. (1965) · Science · doi:10.1126/science.149.3685.754 · Cited by 1,004
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
Virus entry by macropinocytosis
Mercer, J., & Helenius, A. (2009) · Nature Cell Biology · doi:10.1038/ncb0509-510 · Cited by 857
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
Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors
Ferrari, F. K., Samulski, T., Shenk, T., & Samulski, R. J. (1996) · Journal of Virology · doi:10.1128/jvi.70.5.3227-3234.1996 · Cited by 761
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
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
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 622
Systemic delivery of genes to striated muscles using adeno-associated viral vectors
Gregorevic, P., Blankinship, M. J., Allen, J. M., Crawford, R. W., Meuse, L., Miller, D. G., Russell, D. W., & Chamberlain, J. S. (2004) · Nature Medicine · doi:10.1038/nm1085 · Cited by 620
The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy
Xie, Q., Bu, W., Bhatia, S., Hare, J., Somasundaram, T., Azzi, A., & Chapman, M. S. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.162250899 · Cited by 612
Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8
Inagaki, K., Fuess, S., Storm, T. A., Gibson, G. A., Mctiernan, C. F., Kay, M. A., & Nakai, H. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.03.014 · Cited by 610
Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors
Snyder, R. O., Miao, C. H., Patijn, G. A., Spratt, S. K., Danos, O., Nagy, D., Gown, A. M., Winther, B., Meuse, L. A., Cohen, L. K., et al. (1997) · Nature Genetics · doi:10.1038/ng0797-270 · Cited by 579
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
Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis
Fisher, K. J., Gao, G. P., Weitzman, M. D., DeMatteo, R. P., Burda, J. F., & Wilson, J. M. (1996) · Journal of Virology · doi:10.1128/jvi.70.1.520-532.1996 · Cited by 546
Studies of small DNA viruses found in various adenovirus preparations: physical, biological, and immunological characteristics.
HOGGAN, M. D., Blacklow, N. R., & Rowe, W. P. (1966) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.55.6.1467 · Cited by 540
Infectious entry pathway of adeno-associated virus and adeno-associated virus vectors
Bartlett, J. S., Wilcher, R., & Samulski, R. J. (2000) · Journal of Virology · doi:10.1128/jvi.74.6.2777-2785.2000 · Cited by 532 · cited 4×
“The host cell surface receptors are concentrated in a specialized area called clathrincoated pits [106, 110].”
Influential
Self-complementary AAV vectors; advances and applications
McCarty, D. M. (2008) · Molecular Therapy · doi:10.1038/mt.2008.171 · Cited by 515
Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons
Duque, S., Joussemet, B., Riviere, C., Marais, T., Dubreil, L., Douar, A.-M., Fyfe, J., Moullier, P., Colle, M.-A., & Barkats, M. (2009) · Molecular Therapy · doi:10.1038/mt.2009.71 · Cited by 511
Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells.
Hermonat, P. L., & Muzyczka, N. (1984) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.81.20.6466 · Cited by 480
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 480
Infectious Clones and Vectors Derived from Adeno-Associated Virus (AAV) Serotypes Other Than AAV Type 2
Rutledge, E. A., Halbert, C. L., & Russell, D. W. (1998) · Journal of Virology · doi:10.1128/jvi.72.1.309-319.1998 · Cited by 452
Particle Size, Surface Coating, and PEGylation Influence the Biodistribution of Quantum Dots in Living Mice
Schipper, M. L., Iyer, G., Koh, A. L., Cheng, Z., Ebenstein, Y., Aharoni, A., Keren, S., Bentolila, L. A., Li, J., Rao, J., et al. (2008) · Small · doi:10.1002/smll.200800003 · Cited by 451
Integration of Adeno-Associated Virus (AAV) and Recombinant AAV Vectors
McCarty, D. M., Young, S. M., & Samulski, R. J. (2004) · Annual Review of Genetics · doi:10.1146/annurev.genet.37.110801.143717 · Cited by 450
Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver
Nathwani, A. C., Gray, J. T., Ng, C. Y., Zhou, J., Spence, Y., Waddington, S. N., Tuddenham, E. G. D., Kemball‐Cook, G., McIntosh, J., Boon‐Spijker, M., et al. (2005) · Blood · doi:10.1182/blood-2005-10-4035 · Cited by 419
Recombinant Adeno-Associated Virus Serotype 9 Leads to Preferential Cardiac Transduction In Vivo
Pacak, C. A., Mah, C. S., Thattaliyath, B. D., Conlon, T. J., Lewis, M. A., Cloutier, D. E., Zolotukhin, I., Tarantal, A. F., & Byrne, B. J. (2006) · Circulation Research · doi:10.1161/01.res.0000237661.18885.f6 · Cited by 406
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
A viral assembly factor promotes AAV2 capsid formation in the nucleolus
Sonntag, F., Schmidt, K., & Kleinschmidt, J. A. (2010) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1001673107 · Cited by 392
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
Adeno-associated virus (AAV) Rep proteins mediate complex formation between AAV DNA and its integration site in human DNA.
Weitzman, M. D., Kyöstió, S. R., Kotin, R. M., & Owens, R. A. (1994) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.91.13.5808 · Cited by 379
Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo
Wang, Z., Ma, H.-I., Li, J., Sun, L., Zhang, J., & Xiao, X. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302133 · Cited by 367
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
The VP1 capsid protein of adeno-associated virus type 2 is carrying a phospholipase A2 domain required for virus infectivity
Girod, A., Wobus, C. E., Zádori, Z., Ried, M., Leike, K., Tijssen, P., Kleinschmidt, J. A., & Hallek, M. (2002) · Journal of General Virology · doi:10.1099/0022-1317-83-5-973 · Cited by 354
Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy
Mount, J. D., Herzog, R. W., Tillson, D. M., Goodman, S. A., Robinson, N., McCleland, M. L., Bellinger, D., Nichols, T. C., Arruda, V. R., Lothrop, C. D., et al. (2002) · Blood · doi:10.1182/blood.v99.8.2670 · Cited by 339
DNA helicase‐mediated packaging of adeno‐associated virus type 2 genomes into preformed capsids
King, J. A., Dubielzig, R., Grimm, D., & Kleinschmidt, J. A. (2001) · The EMBO Journal · doi:10.1093/emboj/20.12.3282 · Cited by 338
Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy
Yan, Z., Zhang, Y., Duan, D., & Engelhardt, J. F. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.97.12.6716 · Cited by 305
Adeno-Associated Virus Type 2 Capsids with Externalized VP1/VP2 Trafficking Domains Are Generated prior to Passage through the Cytoplasm and Are Maintained until Uncoating Occurs in the Nucleus
Sonntag, F., Bleker, S., Leuchs, B., Fischer, R., & Kleinschmidt, J. A. (2006) · Journal of Virology · doi:10.1128/jvi.01056-06 · Cited by 297
Integration of the adeno-associated virus genome into cellular DNA in latently infected human Detroit 6 cells
Cheung, A. K., HOGGAN, M. D., Hauswirth, W. W., & Berns, K. I. (1980) · Journal of Virology · doi:10.1128/jvi.33.2.739-748.1980 · Cited by 287
A Phase I Study of an Adeno-Associated Virus-CFTR Gene Vector in Adult CF Patients with Mild Lung Disease. Johns Hopkins Children's Center, Baltimore, Maryland
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
Genetic analysis of adeno-associated virus: properties of deletion mutants constructed in vitro and evidence for an adeno-associated virus replication function
Tratschin, J.-D., Miller, I. L., & Carter, B. J. (1984) · Journal of Virology · doi:10.1128/jvi.51.3.611-619.1984 · Cited by 272
Endocytosis and Nuclear Trafficking of Adeno-Associated Virus Type 2 Are Controlled by Rac1 and Phosphatidylinositol-3 Kinase Activation
Şanlıoğlu, S., Benson, P. K., Yang, J., Atkinson, E. M., Reynolds, T. C., & Engelhardt, J. F. (2000) · Journal of Virology · doi:10.1128/jvi.74.19.9184-9196.2000 · Cited by 272
Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle
Asokan, A., Conway, J. C., Phillips, J. L., Li, C., Hegge, J., Sinnott, R., Yadav, S. S., DiPrimio, N., Nam, H. J., Agbandje‐McKenna, M., et al. (2009) · Nature Biotechnology · doi:10.1038/nbt.1599 · Cited by 270
Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes
Halbert, C. L., Rutledge, E. A., Allen, J. M., Russell, D. W., & Miller, A. D. (2000) · Journal of Virology · doi:10.1128/jvi.74.3.1524-1532.2000 · Cited by 259
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 257
Intracellular trafficking of adeno-associated viral vectors
Ding, W., Zhang, L., Yan, Z., & Engelhardt, J. F. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302527 · Cited by 251
Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina
Bennett, J., Maguire, A. M., Cideciyan, A. V., Schnell, M. A., Glover, E., Anand, V., Alemán, T. S., Chirmule, N. B., Gupta, A. R., Huang, Y., et al. (1999) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.96.17.9920 · Cited by 244
Adeno-associated virus Rep proteins target DNA sequences to a unique locus in the human genome
Surosky, R. T., Urabe, M., Godwin, S. G., McQuiston, S. A., Kurtzman, G. J., OZAWA, K., & Natsoulis, G. (1997) · Journal of Virology · doi:10.1128/jvi.71.10.7951-7959.1997 · Cited by 244
Two novel adeno-associated viruses from cynomolgus monkey: pseudotyping characterization of capsid protein
Mori, S., Wang, L., Takeuchi, T., & Kanda, T. (2004) · Virology · doi:10.1016/j.virol.2004.10.012 · Cited by 240
Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors
Yan, Z., Zak, R., Luxton, G. W. G., Ritchie, T. C., Bantel-Schaal, U., & Engelhardt, J. F. (2002) · Journal of Virology · doi:10.1128/jvi.76.5.2043-2053.2002 · Cited by 222
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
Adeno-Associated Virus 2 Infection Requires Endocytosis through the CLIC/GEEC Pathway
Nonnenmacher, M. E., & Weber, T. (2011) · Cell Host & Microbe · doi:10.1016/j.chom.2011.10.014 · Cited by 209
Intramuscular Administration of AAV1-Lipoprotein Lipase S447X Lowers Triglycerides in Lipoprotein Lipase–Deficient Patients
Stroes, E. S., Nierman, M. C., Meulenberg, J. J., Franssen, R., Twisk, J., Henny, C. P., Maas, M. M., Zwinderman, A. H., Ross, C., Aronica, E., et al. (2008) · Arteriosclerosis, Thrombosis, and Vascular Biology · doi:10.1161/atvbaha.108.175620 · Cited by 209
The adeno-associated virus (AAV) Rep protein acts as both a repressor and an activator to regulate AAV transcription during a productive infection
Pereira, D. J., McCarty, D. M., & Muzyczka, N. (1997) · Journal of Virology · doi:10.1128/jvi.71.2.1079-1088.1997 · Cited by 208
Virus-Mediated Transduction of Murine Retina with Adeno-Associated Virus: Effects of Viral Capsid and Genome Size
Yang, G. S., Schmidt, M., Yan, Z., Lindbloom, J. D., Harding, T. C., Donahue, B. A., Engelhardt, J. F., Kotin, R. M., & Davidson, B. L. (2002) · Journal of Virology · doi:10.1128/jvi.76.15.7651-7660.2002 · Cited by 208
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
Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery
Weber, M., Rabinowitz, J., Provost, N., Conrath, H., Folliot, S., Briot, D., Chérel, Y., Chenuaud, P., Samulski, J., Moullier, P., et al. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(03)00098-4 · Cited by 203
Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or -2 serotypes for widespread gene delivery to the neonatal mouse brain
Broekman, M. L. D., Comer, L. A., Hyman, B. T., & Sena‐Esteves, M. (2006) · Neuroscience · doi:10.1016/j.neuroscience.2005.11.057 · Cited by 201
Mutational analysis of narrow pores at the fivefold symmetry axes of adeno-associated virus type 2 capsids reveals a dual role in genome packaging and activation of phospholipase A2 activity
Bleker, S., Sonntag, F., & Kleinschmidt, J. A. (2005) · Journal of Virology · doi:10.1128/jvi.79.4.2528-2540.2005 · Cited by 200 · cited 1×
“This has been attributed to the prevention of virion escape from the endosomes, thus suggesting a major role for the phospholipase domain in this process [43-45].”
Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs
Jiang, H., Lillicrap, D., Patarroyo-White, S. L., Liu, T., Qian, X., Scallan, C. D., Powell, S., Keller, T., McMurray, M., Labelle, A., et al. (2006) · Blood · doi:10.1182/blood-2005-12-5115 · Cited by 200
In vivo gene delivery to the lung using polyethylenimine and fractured polyamidoamine dendrimers
Rudolph, C., Lausier, J., Naundorf, S., M�ller, R. H., & Rosenecker, J. (2000) · The Journal of Gene Medicine · doi:10.1002/1521-2254(200007/08)2:4 · Cited by 194
Structural Proteins of Adenovirus-Associated Viruses
Rose, J. A., Maizel, J. V., Inman, J. K., & Shatkin, A. J. (1971) · Journal of Virology · doi:10.1128/jvi.8.5.766-770.1971 · Cited by 193
Molecular aspects of the endocytic pathway
Clague, M. J. (1998) · Biochemical Journal · doi:10.1042/bj3360271 · Cited by 189
Characterization of the DNA of a defective human parvovirus isolated from a genital site
Bantel‐Schaal, U., & Hausen, H. Z. (1984) · Virology · doi:10.1016/0042-6822(84)90271-x · Cited by 188
Dynamin Is Required for Recombinant Adeno-Associated Virus Type 2 Infection
Duan, D., Li, Q., Kao, A. W., Yue, Y., Pessin, J. E., & Engelhardt, J. F. (1999) · Journal of Virology · doi:10.1128/jvi.73.12.10371-10376.1999 · Cited by 186
Recirculating cardiac delivery of AAV2/1SERCA2a improves myocardial function in an experimental model of heart failure in large animals
Byrne, M. J., Power, J. M., Preovolos, A., Mariani, J. A., Hajjar, R. J., & Kaye, D. M. (2008) · Gene Therapy · doi:10.1038/gt.2008.120 · Cited by 184
EVIDENCE FOR A SINGLE-STRANDED ADENOVIRUS-ASSOCIATED VIRUS GENOME: FORMATION OF A DNA DENSITY HYBRID ON RELEASE OF VIRAL DNA
Rose, J. A., Berns, K. I., Hoggan, M. D., & Koczot, F. J. (1969) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.64.3.863 · Cited by 179
Adeno-associated virus P5 promoter contains an adenovirus E1A-inducible element and a binding site for the major late transcription factor
Chang, L., Shi, Y., & Shenk, T. E. (1989) · Journal of Virology · doi:10.1128/jvi.63.8.3479-3488.1989 · Cited by 178
Adeno-Associated Virus Integration: Virus versus Vector
Smith, R. H. (2008) · Gene Therapy · doi:10.1038/gt.2008.55 · Cited by 178
A largely random AAV integration profile after LPLD gene therapy
Kaeppel, C., Beattie, S. G., Fronza, R., van Logtenstein, R., Salmon, F., Schmidt, S., Wolf, S., Nowrouzi, A., Glimm, H., von Kalle, C., et al. (2013) · Nature Medicine · doi:10.1038/nm.3230 · Cited by 176
Cytoplasmic Trafficking, Endosomal Escape, and Perinuclear Accumulation of Adeno-Associated Virus Type 2 Particles Are Facilitated by Microtubule Network
Xiao, P.-J., & Samulski, R. J. (2012) · Journal of Virology · doi:10.1128/jvi.00935-12 · Cited by 175
Separate basic region motifs within the adeno-associated virus capsid proteins are essential for infectivity and assembly
Grieger, J. C., Snowdy, S., & Samulski, R. J. (2006) · Journal of Virology · doi:10.1128/jvi.02723-05 · Cited by 172 · cited 3×
“The acidification of the capsid, triggers the exposure of N-terminal phospholipase A2 domain and nuclear localization signal of VP1 protein, facilitating their endosomal escape and nuclear entry by traversing the cellular organelles such as golgi and endoplasmic reticulum [42, 46, 116, 117].”
In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector
During, M. J., Samulski, R. J., Elsworth, J. D., Kaplitt, M. G., Leone, P., Xiao, X., Li, J., Freese, A. B., Taylor, J., Rh, R., et al. (1998) · Gene Therapy · doi:10.1038/sj.gt.3300650 · Cited by 171
Enhancement of Adeno-Associated Virus Infection by Mobilizing Capsids into and Out of the Nucleolus
Johnson, J. S., & Samulski, R. J. (2008) · Journal of Virology · doi:10.1128/jvi.02309-08 · Cited by 167
Adeno-Associated Virus Type 12 (AAV12): a Novel AAV Serotype with Sialic Acid- and Heparan Sulfate Proteoglycan-Independent Transduction Activity
Schmidt, M., Voutetakis, A., Afione, S. A., Zheng, C., Mandikian, D., & Chiorini, J. A. (2007) · Journal of Virology · doi:10.1128/jvi.02012-07 · Cited by 162
Identification of linear DNA sequences that specifically bind the adeno-associated virus Rep protein
McCarty, D. M., Pereira, D. J., Zolotukhin, I., Zhou, X., Ryan, J. H., & Muzyczka, N. (1994) · Journal of Virology · doi:10.1128/jvi.68.8.4988-4997.1994 · Cited by 161
Green Fluorescent Protein-Tagged Adeno-Associated Virus Particles Allow the Study of Cytosolic and Nuclear Trafficking
Lux, K., Goerlitz, N., Schlemminger, S., Perabò, L., Goldnau, D., Endell, J., Leike, K., Kofler, D. M., Finke, S., Hallek, M. J., et al. (2005) · Journal of Virology · doi:10.1128/jvi.79.18.11776-11787.2005 · Cited by 159
Phosphorylation of a Novel Myosin Binding Subunit of Protein Phosphatase 1 Reveals a Conserved Mechanism in the Regulation of Actin Cytoskeleton
Tan, I., Ng, C. H., Lim, L., & Leung, T. (2001) · Journal of Biological Chemistry · doi:10.1074/jbc.m102615200 · Cited by 155
Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber’s hereditary optic neuropathy in a mouse model
Yu, H., Koilkonda, R. D., Chou, T.-H., Porciatti, V., Ozdemir, S. S., Chiodo, V., Boye, S. L., Boye, S. E., Hauswirth, W. W., Lewin, A. S., et al. (2012) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1119577109 · Cited by 154
Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose
Wu, Z., Sun, J., Zhang, T., Yin, C., Yin, F., Van Dyke, T., Samulski, R. J., & Monahan, P. E. (2008) · Molecular Therapy · doi:10.1038/sj.mt.6300355 · Cited by 153
Structural Characterization of the Dual Glycan Binding Adeno-Associated Virus Serotype 6
Ng, R., Govindasamy, L., 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 148
Detection of adeno-associated virus (AAV)-specific nucleotide sequences in DNA isolated from latently infected Detroit 6 cells
Berns, K. I., Pinkerton, T. C., Thomas, G., & HOGGAN, M. D. (1975) · Virology · doi:10.1016/0042-6822(75)90298-6 · Cited by 147
Adeno-associated virus Rep78 protein and terminal repeats enhance integration of DNA sequences into the cellular genome
Balagué, C., Kalla, M., & Zhang, W. (1997) · Journal of Virology · doi:10.1128/jvi.71.4.3299-3306.1997 · Cited by 147
Impaired Intracellular Trafficking of Adeno-Associated Virus Type 2 Vectors Limits Efficient Transduction of Murine Fibroblasts
Hansen, J. J., Qing, K., Kwon, H., Mah, C. S., & Srivastava, A. (2000) · Journal of Virology · doi:10.1128/jvi.74.2.992-996.2000 · Cited by 147
A Dual Role of EGFR Protein Tyrosine Kinase Signaling in Ubiquitination of AAV2 Capsids and Viral Second-strand DNA Synthesis
Li, Z., Zhao, W., Wu, J., Li, B., Zolotukhin, S., Govindasamy, L., Agbandje‐McKenna, M., & Srivastava, A. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300170 · Cited by 147
Molecular Basis for Recognition of Dilysine Trafficking Motifs by COPI
Jackson, L. P., Lewis, M., Kent, H. M., Edeling, M. A., Evans, P. R., Duden, R., & Owen, D. J. (2012) · Developmental Cell · doi:10.1016/j.devcel.2012.10.017 · Cited by 146
Transduction of murine cerebellar neurons with recombinant FIV and AAV5 vectors
Alisky, J. M., Hughes, S. M., Sauter, S. L., Jolly, D. J., Dubensky, T. W., Staber, P. D., Chiorini, J. A., & Davidson, B. L. (2000) · Neuroreport · doi:10.1097/00001756-200008210-00013 · Cited by 143
Adenovirus-Facilitated Nuclear Translocation of Adeno-Associated Virus Type 2
Xiao, W. S., Warrington, K. H., Hearing, P., Hughes, J. A., & Muzyczka, N. (2002) · Journal of Virology · doi:10.1128/jvi.76.22.11505-11517.2002 · Cited by 142
Recruitment of wild-type and recombinant adeno-associated virus into adenovirus replication centers
Weitzman, M. D., Fisher, K. J., & Wilson, J. M. (1996) · Journal of Virology · doi:10.1128/jvi.70.3.1845-1854.1996 · Cited by 142
Roles of Adeno-Associated Virus Rep Protein and Human Chromosome 19 in Site-Specific Recombination
Young, S., McCarty, D. M., Degtyareva, N. P., & Samulski, R. J. (2000) · Journal of Virology · doi:10.1128/jvi.74.9.3953-3966.2000 · Cited by 138
A subset of herpes simplex virus replication genes provides helper functions for productive adeno-associated virus replication
Weindler, F. W., & Heilbronn, R. (1991) · Journal of Virology · doi:10.1128/jvi.65.5.2476-2483.1991 · Cited by 137
CNS-directed AAV2-mediated gene therapy ameliorates functional deficits in a murine model of infantile neuronal ceroid lipofuscinosis
Griffey, M. A., Wozniak, D. F., Wong, M. L., Bible, E., Johnson, K. N., Rothman, S. M., Wentz, A. E., Cooper, J. D., & Sands, M. S. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.11.008 · Cited by 137
Systemic AAV6 Delivery Mediating RNA Interference Against SOD1: Neuromuscular Transduction Does Not Alter Disease Progression in fALS Mice
Towne, C. L., Raoul, C., Schneider, B. L., & Aebischer, P. (2008) · Molecular Therapy · doi:10.1038/mt.2008.73 · Cited by 137
Precise hit: adeno-associated virus in gene targeting
Vasileva, A., & Jessberger, R. (2005) · Nature Reviews Microbiology · doi:10.1038/nrmicro1266 · Cited by 135
High levels of persistent expression of α1-antitrypsin mediated by the nonhuman primate serotype rh. 10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses
De, B. P., Heguy, A., Hackett, N. R., Ferris, B., Leopold, P. L., Lee, J., Pierre, L., Gao, G., Wilson, J. M., & Crystal, R. G. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2005.09.003 · Cited by 129
Large-scale recombinant adeno-associated virus production
Kotin, R. M. (2011) · Human Molecular Genetics · doi:10.1093/hmg/ddr141 · Cited by 128
Adeno-associated virus-2 and its primary cellular receptor—Cryo-EM structure of a heparin complex
O’Donnell, J., Taylor, K. A., & Chapman, M. S. (2009) · Virology · doi:10.1016/j.virol.2008.11.037 · Cited by 127
Proteasome Inhibitors Enhance Gene Delivery by AAV Virus Vectors Expressing Large Genomes in Hemophilia Mouse and Dog Models: A Strategy for Broad Clinical Application
Monahan, P. E., Lothrop, C. D., Sun, J., Hirsch, M. L., Kafri, T., Kantor, B., Sarkar, R., Tillson, D. M., Elia, J. R., & Samulski, R. J. (2010) · Molecular Therapy · doi:10.1038/mt.2010.170 · Cited by 125
Adeno-associated virus 2-mediated gene transfer in vivo: organ-tropism and expression of transduced sequences in mice
Ponnazhagan, S., Mukherjee, P., Yöder, M. C., Wang, X.-S., Zhou, S. Z., Kaplan, J. M., Wadsworth, S. C., & Srivastava, A. (1997) · Gene · doi:10.1016/s0378-1119(96)00576-8 · Cited by 125
Structural Proteins of Adenovirus-Associated Virus Type 3
Johnson, F. B., Ozer, H. L., & HOGGAN, M. D. (1971) · Journal of Virology · doi:10.1128/jvi.8.6.860-863.1971 · Cited by 119
Physical Assay and Growth Cycle Studies of a Defective Adeno-Satellite Virus
Parks, W. P., Melnick, J. L., Rongey, R. W., & Mayor, H. D. (1967) · Journal of Virology · doi:10.1128/jvi.1.1.171-180.1967 · Cited by 118
Liver Transduction with Recombinant Adeno-Associated Virus Is Primarily Restricted by Capsid Serotype Not Vector Genotype
Grimm, D., Pandey, K., Nakai, H., Storm, T. A., & Kay, M. A. (2006) · Journal of Virology · doi:10.1128/jvi.80.1.426-439.2006 · Cited by 118
Crystal Structure of the SF3 Helicase from Adeno-Associated Virus Type 2
James, J. A., Escalante, C. R., Yoon-Robarts, M., Edwards, T. A., Linden, R. M., & Aggarwal, A. K. (2003) · Structure · doi:10.1016/s0969-2126(03)00152-7 · Cited by 116
Adeno‐associated virus (AAV) vectors in gene therapy: immune challenges and strategies to circumvent them
Hareendran, S., Balakrishnan, B., Sen, D., Kumar, S., Srivastava, A., & Jayandharan, G. R. (2013) · Reviews in Medical Virology · doi:10.1002/rmv.1762 · Cited by 113
Self-citation
Heparin binding induces conformational changes in Adeno-associated virus serotype 2
Levy, H. C., Bowman, V. D., Govindasamy, L., McKenna, R., Nash, K., Warrington, K., Chen, W., Muzyczka, N., Yan, X., Baker, T. S., et al. (2009) · Journal of Structural Biology · doi:10.1016/j.jsb.2008.12.002 · Cited by 110
Mutagenesis of Adeno-Associated Virus Type 2 Capsid Protein VP1 Uncovers New Roles for Basic Amino Acids in Trafficking and Cell-Specific Transduction
Johnson, J. S., Li, C., DiPrimio, N., Weinberg, M. S., McCown, T. J., & Samulski, R. J. (2010) · Journal of Virology · doi:10.1128/jvi.00687-10 · Cited by 110
Interfacial Enzymology of Parvovirus Phospholipases A2
Canaan, S., Zádori, Z., Ghomashchi, F., Bollinger, J., Sadı́lek, M., Moreau, M. E., Tijssen, P., & Gelb, M. H. (2004) · Journal of Biological Chemistry · doi:10.1074/jbc.m312630200 · Cited by 109
Effect of Adeno-Associated Virus Serotype and Genomic Structure on Liver Transduction and Biodistribution in Mice of Both Genders
Pañeda, A., Vanrell, L., Mauleon, I., Crettaz, J. S., Berraondo, P., Timmermans, E. J., Beattie, S. G., Twisk, J., van Deventer, S., Prieto, J., et al. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.031 · Cited by 109
Enhancing the Clinical Potential of AAV Vectors by Capsid Engineering to Evade Pre-Existing Immunity
Bartel, M. (2011) · Frontiers in Microbiology · doi:10.3389/fmicb.2011.00204 · Cited by 108
Self-Complementary Recombinant Adeno-Associated Viral Vectors: Packaging Capacity And The Role of Rep Proteins in Vector Purity
Wu, J., Zhao, W., Zhong, L., Han, Z., Li, B., Ma, W., Weigel-Kelley, K. A., Warrington, K. H., & Srivastava, A. (2007) · Human Gene Therapy · doi:10.1089/hum.2006.088 · Cited by 108
Identification of a DNA-binding domain in the amino terminus of adeno-associated virus Rep proteins
Owens, R. A., Weitzman, M. D., Kyöstió, S. R., & Carter, B. J. (1993) · Journal of Virology · doi:10.1128/jvi.67.2.997-1005.1993 · Cited by 107
Integration Preferences of Wildtype AAV-2 for Consensus Rep-Binding Sites at Numerous Loci in the Human Genome
Hüser, D., Gogol‐Döring, A., Lutter, T., Weger, S., Winter, K., Hammer, E.-M., Cathomen, T., Reinert, K., & Heilbronn, R. (2010) · PLoS Pathogens · doi:10.1371/journal.ppat.1000985 · Cited by 107
Adeno-Associated Virus Type 5: Transduction Efficiency and Cell-Type Specificity in the Primate Retina
Lotery, A. J., Yang, G. S., Mullins, R. F., Russell, S. R., Schmidt, M., Stone, E. M., Lindbloom, J. D., Chiorini, J. A., Kotin, R. M., & Davidson, B. L. (2003) · Human Gene Therapy · doi:10.1089/104303403322542301 · Cited by 107
Inhibition of S‐phase progression by adeno‐associated virus Rep78 protein is mediated by hypophosphorylated pRb
Saudan, P., Vlach, J., & Beard, P. (2000) · The EMBO Journal · doi:10.1093/emboj/19.16.4351 · Cited by 104
rAAV2 traffics through both the late and the recycling endosomes in a dose-dependent fashion
Ding, W., Zhang, L. N., Yeaman, C. A., & Engelhardt, J. F. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2005.12.002 · Cited by 103
Long-Term Efficacy of Adeno-Associated Virus Serotypes 8 and 9 in Hemophilia A Dogs and Mice
Sarkar, R., Mucci, M., Addya, S., Tetreault, R., Bellinger, D. A., Nichols, T. C., & Kazazian, H. H. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.427 · Cited by 102
Sequence requirements for stable binding and function of Rep68 on the adeno-associated virus type 2 inverted terminal repeats
Chiorini, J. A., Wiener, S. M., Owens, R. A., Kyöstió, S. R., Kotin, R. M., & Safer, B. (1994) · Journal of Virology · doi:10.1128/jvi.68.11.7448-7457.1994 · Cited by 101
Zinc-Finger Nuclease Based Genome Surgery: Its All About Specificity
Händel, E.-M., & Cathomen, T. (2011) · Current Gene Therapy · doi:10.2174/156652311794520120 · Cited by 99
Molecular Characterization of Adeno-Associated Viruses Infecting Children
Chen, C., Jensen, R. L., Schnepp, B. C., Connell, M. J., SHELL, R. D., Sferra, T. J., Bartlett, J. S., Clark, K. R., & Johnson, P. R. (2005) · Journal of Virology · doi:10.1128/jvi.79.23.14781-14792.2005 · Cited by 99
How adeno-associated virus Rep78 protein arrests cells completely in S phase
Berthet, C., Raj, K., Saudan, P., & Beard, P. (2005) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0504583102 · Cited by 97
Bioengineering of AAV2 Capsid at Specific Serine, Threonine, or Lysine Residues Improves Its Transduction Efficiency in Vitro and in Vivo
Gabriel, N., Hareendran, S., Sen, D., Gadkari, R. A., Sudha, G., Selot, R., Hussain, M., Dhaksnamoorthy, R., Samuel, R., Srinivasan, N., et al. (2013) · Human Gene Therapy Methods · doi:10.1089/hgtb.2012.194 · Cited by 92
Self-citation
Adeno-associated virus site-specifically integrates into a muscle-specific DNA region
Dutheil, N., Shi, F., Dupressoir, T., & Linden, R. M. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.080079397 · Cited by 92
Negative regulation of the adeno-associated virus (AAV) P5 promoter involves both the P5 rep binding site and the consensus ATP-binding motif of the AAV Rep68 protein
Kyöstió, S. R., Wonderling, R. S., & Owens, R. A. (1995) · Journal of Virology · doi:10.1128/jvi.69.11.6787-6796.1995 · Cited by 90
Enhanced Real-Time Monitoring of Adeno-Associated Virus Trafficking by Virus–Quantum Dot Conjugates
Joo, K., Fang, Y. H., Liu, Y., Liang, X., Gu, Z., Tai, A., Lee, C.-L., Tang, Y., & Wang, P. (2011) · ACS Nano · doi:10.1021/nn102651p · Cited by 89
Differential Myocardial Gene Delivery by Recombinant Serotype-Specific Adeno-associated Viral Vectors
Du, L., Kido, M., Lee, D. V., Rabinowitz, J. E., Samulski, R. J., Jamieson, S. W., Weitzman, M. D., & Thistlethwaite, P. A. (2004) · Molecular Therapy · doi:10.1016/j.ymthe.2004.06.110 · Cited by 86
Mechanism of Rep-Mediated Adeno-Associated Virus Origin Nicking
Brister, J. R., & Muzyczka, N. (2000) · Journal of Virology · doi:10.1128/jvi.74.17.7762-7771.2000 · Cited by 86
Zinc-finger nuclease-mediated correction of α-thalassemia in iPS cells
Chang, C.-J., & Bouhassira, E. E. (2012) · Blood · doi:10.1182/blood-2012-03-420703 · Cited by 84
Role of the Adenovirus DNA-Binding Protein in In Vitro Adeno-Associated Virus DNA Replication
Ward, P., Dean, F. B., O’Donnell, M. E., & Berns, K. I. (1998) · Journal of Virology · doi:10.1128/jvi.72.1.420-427.1998 · Cited by 83
Adenovirus E1B 55-Mr polypeptide facilitates timely cytoplasmic accumulation of adeno-associated virus mRNAs
Samulski, R. J., & Shenk, T. E. (1988) · Journal of Virology · doi:10.1128/jvi.62.1.206-210.1988 · Cited by 82
Identification of Cellular Proteins That Interact with the Adeno-Associated Virus Rep Protein
Nash, K. R., Chen, W., Salganik, M., & Muzyczka, N. (2008) · Journal of Virology · doi:10.1128/jvi.01939-08 · Cited by 82
Evidence for pH-Dependent Protease Activity in the Adeno-Associated Virus Capsid
Salganik, M., Venkatakrishnan, B., Bennett, A. D., Lins, B., Yarbrough, J., Muzyczka, N., Agbandje‐McKenna, M., & McKENNA, R. V. (2012) · Journal of Virology · doi:10.1128/jvi.01717-12 · Cited by 80
High-Level Transgene Expression in Nonhuman Primate Liver with Novel Adeno-Associated Virus Serotypes Containing Self-Complementary Genomes
Gao, G., Lü, Y., Sun, X., Johnston, J., Calcedo, R., Grant, R. L., & Wilson, J. M. (2006) · Journal of Virology · doi:10.1128/jvi.00526-06 · Cited by 77
Long-Term Correction of Ammonia Metabolism and Prolonged Survival in Ornithine Transcarbamylase-Deficient Mice Following Liver-Directed Treatment with Adeno-associated Viral Vectors
Moscioni, D., Morizono, H., McCarter, R., Stern, A., Cabrera‐Luque, J., Hoang, A., Sanmiguel, J. C., Wu, D., Bell, P. L., Gao, G., et al. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.03.009 · Cited by 76
Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate
Colle, M.-A., Piguet, F., Bertrand, L., Raoul, S., Bièche, I., Dubreil, L., Sloothaak, D., Bouquet, C., Moullier, P., Aubourg, P. R., et al. (2009) · Human Molecular Genetics · doi:10.1093/hmg/ddp475 · Cited by 73
Site-specific integration of adeno-associated virus involves partial duplication of the target locus
Henckaerts, E., Dutheil, N., Zeltner, N., Kattman, S. J., Kohlbrenner, E., Ward, P., Clément, N., Rebollo, P., Kennedy, M., Keller, G. M., et al. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0806821106 · Cited by 72
Adeno-associated Virus: A Key to the Human Genome?
Henckaerts, E., & Linden, R. M. (2010) · Future Virology · doi:10.2217/fvl.10.48 · Cited by 71
AAV9-mediated erythropoietin gene delivery into the brain protects nigral dopaminergic neurons in a rat model of Parkinson's disease
Xue, Y.-Q., Ma, B.-F., Zhao, L.-R., Tatom, J. B., Li, B., Jiang, L.-X., Klein, R. L., & Duan, W.-M. (2009) · Gene Therapy · doi:10.1038/gt.2009.113 · Cited by 70
The Role of DNA-PKcs and Artemis in Opening Viral DNA Hairpin Termini in Various Tissues in Mice
Inagaki, K., Ma, C., Storm, T. A., Kay, M. A., & Nakai, H. (2007) · Journal of Virology · doi:10.1128/jvi.01225-07 · Cited by 70
Viral serotype and the transgene sequence influence overlapping adeno‐associated viral (AAV) vector‐mediated gene transfer in skeletal muscle
Ghosh, A., Yue, Y., & Duan, D. (2006) · The Journal of Gene Medicine · doi:10.1002/jgm.835 · Cited by 69
Complete In Vitro Reconstitution of Adeno-Associated Virus DNA Replication Requires the Minichromosome Maintenance Complex Proteins
Nash, K. R., Chen, W., & Muzyczka, N. (2007) · Journal of Virology · doi:10.1128/jvi.01968-07 · Cited by 68
Redundant and Synergistic Mechanisms Control the Sequestration of Blood-born Adenovirus in the Liver
Paolo, N. C. D., van Rooijen, N., & Shayakhmetov, D. M. (2009) · Molecular Therapy · doi:10.1038/mt.2008.307 · Cited by 67
The Rep Protein of Adeno-Associated Virus Type 2 Interacts with Single-Stranded DNA-Binding Proteins That Enhance Viral Replication
Stracker, T. H., Cassell, G. D., Ward, P., Loo, Y., van Breukelen, B., Carrington-Lawrence, S. D., Hamatake, R. K., van der Vliet, P. C., Weller, S. K., Melendy, T., et al. (2003) · Journal of Virology · doi:10.1128/jvi.78.1.441-453.2004 · Cited by 67
Efficient and long-term intracardiac gene transfer in δ-sarcoglycan-deficiency hamster by adeno-associated virus-2 vectors
Li, J., Wang, D., Qian, S., Chen, Z., Zhu, T., & Xiao, X. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302078 · Cited by 63
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 61
Dual Reporter Comparative Indexing of rAAV Pseudotyped Vectors in Chimpanzee Airway
Flotte, T. R., Fischer, A. C., Goetzmann, J. E., Mueller, C., Cebotaru, L., Yan, Z., Wang, L., Wilson, J. M., Guggino, W. B., & Engelhardt, J. F. (2009) · Molecular Therapy · doi:10.1038/mt.2009.230 · Cited by 56
Lipofection of Purified Adeno-Associated Virus Rep68 Protein: toward a Chromosome-Targeting Nonviral Particle
Lamartina, S., Roscilli, G., Rinaudo, D., Delmastro, P., & Toniatti, C. (1998) · Journal of Virology · doi:10.1128/jvi.72.9.7653-7658.1998 · Cited by 56
Residues within the B′ Motif Are Critical for DNA Binding by the Superfamily 3 Helicase Rep40 of Adeno-associated Virus Type 2
Yoon-Robarts, M., Blouin, A. G., Bleker, S., Kleinschmidt, J. A., Aggarwal, A. K., Escalante, C. R., & Linden, R. M. (2004) · Journal of Biological Chemistry · doi:10.1074/jbc.m403900200 · Cited by 52
Adeno-Associated Virus Site-Specific Integration Is Mediated by Proteins of the Nonhomologous End-Joining Pathway
Daya, S., Cortez, N. G., & Berns, K. I. (2009) · Journal of Virology · doi:10.1128/jvi.01040-09 · Cited by 52
Targeted Modifications in Adeno-Associated Virus Serotype 8 Capsid Improves Its Hepatic Gene Transfer Efficiency In Vivo
Sen, D., Gadkari, R. A., Sudha, G., Gabriel, N., Kumar, Y. S., Selot, R., Samuel, R., Rajalingam, S., Ramya, V., Nair, S. C., et al. (2013) · Human Gene Therapy Methods · doi:10.1089/hgtb.2012.195 · Cited by 51
Self-citation
Activation of the Cellular Unfolded Protein Response by Recombinant Adeno-Associated Virus Vectors
Balakrishnan, B., Sen, D., Hareendran, S., Roshini, V., David, S., Srivastava, A., & Jayandharan, G. R. (2013) · PLoS ONE · doi:10.1371/journal.pone.0053845 · Cited by 50
Self-citation
AAV Exploits Subcellular Stress Associated with Inflammation, Endoplasmic Reticulum Expansion, and Misfolded Proteins in Models of Cystic Fibrosis
Johnson, J. S., Gentzsch, M., Zhang, L., Ribeiro, C. M. P., Kantor, B., Kafri, T., Pickles, R. J., & Samulski, R. J. (2011) · PLoS Pathogens · doi:10.1371/journal.ppat.1002053 · Cited by 49
Atomic structure of viral particles
Chapman, M. S., & Agbandje‐McKenna, M. (2005) · CRC Press eBooks · doi:10.1201/b13393-13 · Cited by 49
Unique Biologic Properties of Recombinant AAV1 Transduction in Polarized Human Airway Epithelia
Yan, Z., Lei-Butters, D. C. M., Liu, X., Zhang, Y., Zhang, L. N., Luo, M., Žák, R., & Engelhardt, J. F. (2006) · Journal of Biological Chemistry · doi:10.1074/jbc.m604099200 · Cited by 48
Improved adeno-associated virus (AAV) serotype 1 and 5 vectors for gene therapy
Sen, D., Balakrishnan, B., Gabriel, N., Agrawal, P. P., Roshini, V., Samuel, R., Srivastava, A., & Jayandharan, G. R. (2013) · Scientific Reports · doi:10.1038/srep01832 · Cited by 44
Self-citation
A Biochemical Characterization of the Adeno-associated Virus Rep40 Helicase
Collaco, R. F., Kalman‐Maltese, V., Smith, A. D., Dignam, J. D., & Trempe, J. P. (2003) · Journal of Biological Chemistry · doi:10.1074/jbc.m301537200 · Cited by 42
Genome Engineering with TAL-Effector Nucleases and Alternative Modular Nuclease Technologies
Scharenberg, A. M., Duchateau, P. N., & Smith, J. N. P. (2013) · Current Gene Therapy · doi:10.2174/15665232113139990026 · Cited by 41
Strategies for improving the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo
Jayandharan, G. R., Zhong, L., Li, B., Kachniarz, B., & Srivastava, A. (2008) · Gene Therapy · doi:10.1038/gt.2008.89 · Cited by 37
Self-citation
γ-Rays enhance rAAV-mediated transgene expression and cytocidal effect of AAV-HSVtk/ganciclovir on cancer cells
Kanazawa, T., Urabe, M., Mizukami, H., Okada, T., Kume, A., Nishino, H., Monahan, J., Kitamura, K., Ichimura, K., & Ozawa, K. (2001) · Cancer Gene Therapy · doi:10.1038/sj.cgt.7700282 · Cited by 35
Adeno-Associated Viruses Can Induce Phosphorylation of eIF2α via PKR Activation, Which Can Be Overcome by Helper Adenovirus Type 5 Virus-Associated RNA
Nayak, R., & Pintel, D. J. (2007) · Journal of Virology · doi:10.1128/jvi.01132-07 · Cited by 33
Targeted Genomic Integration of a Selectable Floxed Dual Fluorescence Reporter in Human Embryonic Stem Cells
Gantz, J. A., Palpant, N. J., Welikson, R. E., Hauschka, S. D., Murry, C. E., & Laflamme, M. A. (2012) · PLoS ONE · doi:10.1371/journal.pone.0046971 · Cited by 33
Conditional Site-Specific Integration into Human Chromosome 19 by Using a Ligand-Dependent Chimeric Adeno-Associated Virus/Rep Protein
Rinaudo, D., Lamartina, S., Roscilli, G., Ciliberto, G., & Toniatti, C. (2000) · Journal of Virology · doi:10.1128/jvi.74.1.281-294.2000 · Cited by 33
Enhanced Long-Term Transduction and Multilineage Engraftment of Human Hematopoietic Stem Cells Transduced with Tyrosine-Modified Recombinant Adeno-Associated Virus Serotype 2
Kauss, M., Smith, L. J., Zhong, L., Srivastava, A., Wong, K. K., & Chatterjee, S. (2010) · Human Gene Therapy · doi:10.1089/hum.2010.016 · Cited by 30
Evidence of Chromosomal Integration of AAV DNA in Human Testis Tissue
Mehrle, S., Rohde, V., & Schlehofer, J. R. (2004) · Virus Genes · doi:10.1023/b:viru.0000012264.54212.f5 · Cited by 30
Characterization of the Mouse Adeno-Associated Virus AAVS1 Ortholog
Dutheil, N., Yoon-Robarts, M., Ward, P., Henckaerts, E., Skrabanek, L. A., Berns, K. I., Campagne, F., & Linden, R. M. (2004) · Journal of Virology · doi:10.1128/jvi.78.16.8917-8921.2004 · Cited by 29
Oligomeric Properties of Adeno-Associated Virus Rep68 Reflect Its Multifunctionality
Zárate-Pérez, F., Mansilla‐Soto, J., Bardelli, M., Burgner, J. W., Villamil-Jarauta, M., Kekilli, D., Samsó, M., Linden, R. M., & Escalante, C. R. (2012) · Journal of Virology · doi:10.1128/jvi.02441-12 · Cited by 29
Studies of the Mechanism of Transactivation of the Adeno-Associated Virus p19 Promoter by Rep Protein
Lackner, D. F., & Muzyczka, N. (2002) · Journal of Virology · doi:10.1128/jvi.76.16.8225-8235.2002 · Cited by 28
Efficient Generation and Amplification of High-Capacity Adeno-Associated Virus/Adenovirus Hybrid Vectors
Gonçalves, M. A. F. V., van der Velde, I., Janssen, J. M., Maassen, B., Heemskerk, E., Opstelten, D.-J. E., Knaän‐Shanzer, S., Valerio, D., & de Vries, A. A. F. (2002) · Journal of Virology · doi:10.1128/jvi.76.21.10734-10744.2002 · Cited by 26
Persistent liver expression of murine apoA-l using vectors based on adeno-associated viral vectors serotypes 5 and 1
Kitajima, K., Marchadier, D. H. L., Burstein, H., & Rader, D. J. (2005) · Atherosclerosis · doi:10.1016/j.atherosclerosis.2005.07.009 · Cited by 24
Analysis of site‐specific transgene integration following cotransduction with recombinant adeno‐associated virus and a rep encodingplasmid
Huttner, N. A., Girod, A., Schnittger, S., Schoch, C., Hallek, M. J., & Büning, H. (2002) · The Journal of Gene Medicine · doi:10.1002/jgm.324 · Cited by 24
Herpes Simplex Virus Type 1 ICP0 Protein Mediates Activation of Adeno-Associated Virus Type 2 rep Gene Expression from a Latent Integrated Form
Geoffroy, M., Epstein, A. L., Toublanc, E., Moullier, P., & Salvetti, A. (2004) · Journal of Virology · doi:10.1128/jvi.78.20.10977-10986.2004 · Cited by 23
Advances in Recombinant Adeno-Associated Viral Vectors for Gene Delivery
Petrs‐Silva, H., & Linden, R. (2013) · Current Gene Therapy · doi:10.2174/15665232113136660028 · Cited by 23
The transient expression of mRNA coding for Rep protein from AAV facilitates targeted plasmid integration
Howden, S. E., Voullaire, L. E., & Vadolas, J. (2007) · The Journal of Gene Medicine · doi:10.1002/jgm.1118 · Cited by 21
Adeno-Associated Virus Type 2 p5 Promoter: a Rep-Regulated DNA Switch Element Functioning in Transcription, Replication, and Site-Specific Integration
Murphy, M., Gomos‐Klein, J., Stankic, M., & Falck-Pedersen, E. (2007) · Journal of Virology · doi:10.1128/jvi.02693-06 · Cited by 16
Site-Specific Integration by the Adeno-Associated Virus Rep Protein
Recchia, A., & Mavilio, F. (2011) · Current Gene Therapy · doi:10.2174/156652311797415809 · Cited by 11
Stable Producer Cell Lines for Adeno-Associated Virus (AAV) AssemblyPreprint
Chadeuf, G., & Salvetti, A. (2010) · Cold Spring Harbor Protocols · doi:10.1101/pdb.prot5496 · Cited by 10
DNA-Binding Activity of Adeno-Associated Virus Rep Is Required for Inverted Terminal Repeat-Dependent Complex Formation with Herpes Simplex Virus ICP8
Alex, M., Weger, S., Mietzsch, M., Slanina, H., Cathomen, T., & Heilbronn, R. (2012) · Journal of Virology · doi:10.1128/jvi.06364-11 · Cited by 9

Sorted by citations

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
Emerging issues in AAV-mediated in vivo gene therapy
Colella, P., Ronzitti, G., & Mingozzi, F. (2018) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2017.11.007 · Cited by 881
Adenovirus-mediated gene delivery: Potential applications for gene and cell-based therapies in the new era of personalized medicine
Lee, C. S., Bishop, E. S., Zhang, R., Yu, X., Farina, E. M., Yan, S., Zhao, C., Zeng, Z., Shu, Y., Wu, X., et al. (2017) · Genes & Diseases · doi:10.1016/j.gendis.2017.04.001 · Cited by 691
AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer
Costa Verdera, H., Kuranda, K., & Mingozzi, F. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2019.12.010 · Cited by 641
Human Immune Responses to Adeno-Associated Virus (AAV) Vectors
Ronzitti, G., Gross, D.-A., & Mingozzi, F. (2020) · Frontiers in Immunology · doi:10.3389/fimmu.2020.00670 · Cited by 357

See all 225

Authors: Balakrishnan, B., & Jayandharan, G.
Affiliations: Bentham Science Publishers (United Arab Emirates) (AE)
Christian Medical College, Vellore (IN)
ORCID: Balakrishnan, Balaji
Jayandharan, Giridhara
Venue: Current Gene Therapy
Publisher: Bentham Science Publishers Ltd.
Published: 2014-05-31
Volume: 14
Issue: 2
Pages: 86-100
ISSN: 1566-5232 (ISSN-L)
DOI: 10.2174/1566523214666140302193709
OpenAlex: W2142695353
Cited by: 225
References: 186
Citations by year: 2026: 6 · 2025: 29 · 2024: 22 · 2023: 24 · 2022: 24 · 2021: 28 · 2020: 37 · 2019: 18 · 2018: 13 · 2017: 13 · 2016: 7 · 2015: 1 · 2014: 3
FWCI: 5.24
Type: journal article
Language: en
Open access: closed
Locations: Current Gene Therapy (publishedVersion)
PubMed (publishedVersion)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Immunology and Microbiology › Immunology › Immunotherapy and Immune Responses
Keywords: adeno-associated virus, adeno-associated virus vectors, gene therapy vectors, host immune response, vector immunogenicity, vector design
MeSH terms: Dependovirus, Genetic Vectors, Humans, Genetic Therapy, Clinical Trials, Phase I as Topic, Clinical Trials, Phase II as Topic, Gene Transfer Techniques, Host-Pathogen Interactions

About these checks

Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.

Statistics and arithmetic are recomputed, not judged.

Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.

What counts as a claim ›How we measure confidence ›How we check this ›

Are you an author, or do you see a problem with this page? Request a review

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

Abstract

Overview

Integrity signals

Verification

Claims the paper makes

Claims other papers make about this one

Citations

Cites

Cited by

Related

Metadata

Record

About these checks