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

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 · see citing works

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

Maguire, A. M., Simonelli, F., Pierce, E. A., Pugh, E. N., Mingozzi, F., Bennicelli, J., Banfi, S., Marshall, K. A., Testa, F., Surace, E. M., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802315 · Cited by 2,145 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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

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

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× · 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

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 · see citing works

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

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

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 · see citing works

Effect of genome size on AAV vector packaging

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

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 · see citing works

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

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

Virus entry by macropinocytosis

Mercer, J., & Helenius, A. (2009) · Nature Cell Biology · doi:10.1038/ncb0509-510 · Cited by 814 · 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

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 · see citing works

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

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

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

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

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 · see citing works

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 · see citing works

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 · see citing works

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

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

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 · see citing works

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

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

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 · see citing works

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 · see citing works

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× · see citing works

Self-complementary AAV vectors; advances and applications

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

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 · see citing works

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 · see citing works

The AAV Vector Toolkit: Poised at the Clinical Crossroads

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

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 · see citing works

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 452 · see citing works

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 · see citing works

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 421 · see citing works

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 413 · see citing works

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 · 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

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 · 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

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 · 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

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 277 · see citing works

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 · see citing works

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 269 · see citing works

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 265 · see citing works

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 · 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

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 · see citing works

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 · see citing works

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 242 · see citing works

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 · see citing works

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 · 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

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 · see citing works

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 · 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

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 · see citing works

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 · see citing works

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× · see citing works

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 200 · see citing works

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 · see citing works

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 199 · see citing works

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., Kalle, C. V., et al. (2013) · Nature Medicine · doi:10.1038/nm.3230 · Cited by 190 · see citing works

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 190 · see citing works

Molecular aspects of the endocytic pathway

Clague, M. J. (1998) · Biochemical Journal · doi:10.1042/bj3360271 · Cited by 189 · see citing works

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 · see citing works

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. A., Boye, S. L., Boye, S. L., Hauswirth, W. W., Lewin, A. S., et al. (2012) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1119577109 · Cited by 187 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

Adeno-Associated Virus Integration: Virus versus Vector

Smith, R. H. (2008) · Gene Therapy · doi:10.1038/gt.2008.55 · Cited by 178 · see citing works

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 175 · see citing works

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× · see citing works

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 · see citing works

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 163 · see citing works

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 · see citing works

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 · see citing works

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 158 · see citing works

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 · see citing works

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 153 · see citing works

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 · see citing works

Precise hit: adeno-associated virus in gene targeting

Vasileva, A., & Jessberger, R. (2005) · Nature Reviews Microbiology · doi:10.1038/nrmicro1266 · Cited by 148 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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., Lakshmanan Govindasamy, A., Agbandje‐McKenna, M., & Srivastava, A. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300170 · Cited by 146 · see citing works

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 · see citing works

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 · see citing works

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

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

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 140 · see citing works

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 · see citing works

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 · see citing works

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 137 · see citing works

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 136 · see citing works

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 129 · see citing works

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 · see citing works

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 126 · see citing works

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 · see citing works

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 · see citing works

Large-scale recombinant adeno-associated virus production

Kotin, R. M. (2011) · Human Molecular Genetics · doi:10.1093/hmg/ddr141 · Cited by 123 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 105 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 101 · see citing works

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 101 · see citing works

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 99 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 90 · see citing works

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 88 · see citing works

Identification of Cellular Proteins That Interact with the Adeno-Associated Virus Rep Protein

Nash, K., Chen, W.-J., Salganik, M., & Muzyczka, N. (2008) · Journal of Virology · doi:10.1128/jvi.01939-08 · Cited by 86 · see citing works

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 · see citing works

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 · see citing works

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 84 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 78 · see citing works

The Role of DNA-PKcs and Artemis in Opening Viral DNA Hairpin Termini in Various Tissues in Mice

Inagaki, K., Ma, C.-R., Storm, T. A., Kay, M., & Nakai, H. (2007) · Journal of Virology · doi:10.1128/jvi.01225-07 · Cited by 76 · see citing works

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 75 · see citing works

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 72 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · 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

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 · see citing works

Multiply deleted [E1, polymerase-, and pTP-] adenovirus vector persists despite deletion of the preterminal protein

Hodges, B. L., Serra, D. M., Hu, H., Begy, C. R., Chamberlain, J. S., & Amalfitano, A. (2000) · The Journal of Gene Medicine · doi:10.1002/1521-2254(200007/08)2:4 · Cited by 55 · see citing works

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 · see citing works

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 · see citing works

Targeted Modifications in Adeno-Associated Virus Serotype 8 Capsid Improves Its Hepatic Gene Transfer Efficiency <i>In Vivo</i>

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 · see citing works

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 48 · see citing works

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 47 · see citing works

Atomic structure of viral particles

Chapman, M. S., & Agbandje‐McKenna, M. (2005) · CRC Press eBooks · doi:10.1201/b13393-13 · Cited by 47 · see citing works

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. (2013) · PLoS ONE · doi:10.1371/journal.pone.0053845 · Cited by 44 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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 · see citing works

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

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

Emerging issues in AAV-mediated in vivo gene therapy

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

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 &amp; Diseases · doi:10.1016/j.gendis.2017.04.001 · Cited by 691 · see citing works

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 · see citing works

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 citing works

Overcoming innate immune barriers that impede AAV gene therapy vectors

Muhuri, M., Maeda, Y., Ma, H., Ram, S., Fitzgerald, K. A., Tai, P. W. L., & Gao, G. (2021) · Journal of Clinical Investigation · doi:10.1172/jci143780 · Cited by 212 · see citing works

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

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

Adeno-associated viral vector-mediated immune responses: Understanding barriers to gene delivery

Nidetz, N. F., McGee, M. C., Tse, L. V., Li, C., Cong, L., Li, Y., & Huang, W. (2020) · Pharmacology &amp; Therapeutics · doi:10.1016/j.pharmthera.2019.107453 · Cited by 174 · see citing works

Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis

Yang, Y.-S., Xie, J., Wang, D., Kim, J., Tai, P. W. L., Gravallese, E. M., Gao, G., & Shim, J. (2019) · Nature Communications · doi:10.1038/s41467-019-10809-6 · Cited by 139 · see citing works

Adeno-associated virus in the liver: natural history and consequences in tumour development

Bella, T. L., Imbeaud, S., Péneau, C., Mami, I., Datta, S., Bayard, Q., Caruso, S., Hirsch, T. Z., Caldéraro, J., Morcrette, G., et al. (2019) · Gut · doi:10.1136/gutjnl-2019-318281 · Cited by 129 · see citing works

Journey to the Center of the Cell: Tracing the Path of AAV Transduction

Dhungel, B. P., Bailey, C. G., & Rasko, J. E. J. (2021) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2020.09.010 · Cited by 122 · see citing works

Adeno-associated Virus Vectors in Gene Therapy

Mary, B., Khan, N., Arumugam, S., Saxena, H., Kumar, M., Manimaran, P., Chattopadhyay, S., & Jayandharan, G. R. (2018) · Springer eBooks · doi:10.1007/978-981-13-0481-1_2 · Cited by 124 · see citing works

Immunogenicity assessment of AAV-based gene therapies: An IQ consortium industry white paper

Yang, T., Braun, M., Lembke, W., McBlane, F., Kamerud, J., DeWall, S. L., Tarcsa, E., Fang, X., Hofer, L., Kavita, U., et al. (2022) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2022.07.018 · Cited by 118 · see citing works

Post‐translational modifications in capsid proteins of recombinant adeno‐associated virus ( AAV ) 1‐rh10 serotypes

Mary, B., Maurya, S., Arumugam, S., Kumar, V. S., & Jayandharan, G. R. (2019) · FEBS Journal · doi:10.1111/febs.15013 · Cited by 102 · see citing works

Nanocarrier vaccines for SARS-CoV-2

Machhi, J., Shahjin, F., Das, S., Patel, M., Abdelmoaty, M. M., Cohen, J. D., Singh, P., Baldi, A., Bajwa, N., Kumar, R., et al. (2021) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2021.01.002 · Cited by 80 · see citing works

Approved and marketed nanoparticles for disease targeting and applications in COVID-19

Abdellatif, A. A. H., & Alsowinea, A. F. (2021) · Nanotechnology Reviews · doi:10.1515/ntrev-2021-0115 · Cited by 101 · see citing works

Efficient gene delivery into cell lines and stem cells using baculovirus

Sung, L., Chen, C.-L., Lin, S.-Y., Li, K.-C., Yeh, C.-L., Chen, G., Lin, C., & Hu, Y. C. (2014) · Nature Protocols · doi:10.1038/nprot.2014.130 · Cited by 92 · see citing works

Dissecting the Role of Subtypes of Gastrointestinal Vagal Afferents

Wang, Y. B., de Lartigue, G., & Page, A. J. (2020) · Frontiers in Physiology · doi:10.3389/fphys.2020.00643 · Cited by 92 · see citing works

Manufacturing of recombinant adeno-associated viruses using mammalian expression platforms

Robert, M., Chahal, P. S., Audy, A., Kamen, A., Gilbert, R., & Gaillet, B. (2017) · Biotechnology Journal · doi:10.1002/biot.201600193 · Cited by 89 · see citing works

Clinical development on the frontier: gene therapy for duchenne muscular dystrophy

Asher, D. R., Thapa, K., Dharia, S. D., Khan, N., Potter, R. A., Rodino‐Klapac, L. R., & Mendell, J. R. (2020) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2020.1725469 · Cited by 90 · see citing works

Strategies for Vaccination: Conventional Vaccine Approaches Versus New-Generation Strategies in Combination with Adjuvants

Bouazzaoui, A., Abdellatif, A. A. H., Al-Allaf, F. A., Bogari, N. M., Al-Dehlawi, S., & Qari, S. H. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13020140 · Cited by 91 · see citing works

Gene Therapy for Regenerative Medicine

Hosseinkhani, H., Domb, A. J., Sharifzadeh, G., & Nahum, V. (2023) · Pharmaceutics · doi:10.3390/pharmaceutics15030856 · Cited by 88 · see citing works

The Different Faces of Rolling-Circle Replication and Its Multifunctional Initiator Proteins

Wawrzyniak, P., Płucienniczak, G., & Bartosik, D. (2017) · Frontiers in Microbiology · doi:10.3389/fmicb.2017.02353 · Cited by 84 · see citing works

Harnessing nanoparticles for the efficient delivery of the CRISPR/Cas9 system

Rahimi, H., Salehiabar, M., Charmi, J., Barsbay, M., Ghaffarlou, M., Razlighi, M. R., Davaran, S., Khalilov, R. I., Sugiyama, M., Nosrati, H., et al. (2020) · Nano Today · doi:10.1016/j.nantod.2020.100895 · Cited by 83 · see citing works

Muscle Atrophy: Present and Future

Cao, R. Y., Li, J., Dai, Q., Li, Q. X., & Yang, J. (2018) · Advances in experimental medicine and biology · doi:10.1007/978-981-13-1435-3_29 · Cited by 75 · see citing works

Adeno-Associated Viruses (AAV) and Host Immunity – A Race Between the Hare and the Hedgehog

Rapti, K., & Grimm, D. (2021) · Frontiers in Immunology · doi:10.3389/fimmu.2021.753467 · Cited by 70 · see citing works

Automated Glycan Assembly of Oligo-N-Acetyllactosamine and Keratan Sulfate Probes to Study Virus-Glycan Interactions

Hahm, H. S., Broecker, F., Kawasaki, F., Mietzsch, M., Heilbronn, R., Fukuda, M., & Seeberger, P. H. (2017) · Chem · doi:10.1016/j.chempr.2016.12.004 · Cited by 70 · see citing works

Evolving AAV-delivered therapeutics towards ultimate cures

He, X., Urip, B. A., Zhang, Z., Ngan, C. C., & Feng, B. (2021) · Journal of Molecular Medicine · doi:10.1007/s00109-020-02034-2 · Cited by 67 · see citing works

Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients

Gross, D., Tedesco, N., Leborgne, C., & Ronzitti, G. (2022) · Frontiers in Immunology · doi:10.3389/fimmu.2022.857276 · Cited by 63 · see citing works

Promise and problems associated with the use of recombinant AAV for the delivery of anti-HIV antibodies

Fuchs, S. P., & Desrosiers, R. C. (2016) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2016.68 · Cited by 61 · see citing works

Adenoviral vectors for in vivo delivery of CRISPR-Cas gene editors

Rice-Boucher, P. J., Cui, X., & Curiel, D. T. (2020) · Journal of Controlled Release · doi:10.1016/j.jconrel.2020.09.003 · Cited by 60 · see citing works

Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development

Massaro, G., Geard, A. F., Liu, W., Coombe-Tennant, O., Waddington, S. N., Baruteau, J., Gissen, P., & Rahim, A. A. (2021) · Biomolecules · doi:10.3390/biom11040611 · Cited by 60 · see citing works

Gene-Based Therapeutics for Inherited Retinal Diseases

Fenner, B. J., Tan, T., Barathi, V. A., Tun, S. B. B., Yeo, S. W., Tsai, A. S. H., Lee, S. Y., Cheung, C. M. G., Chan, C. M., Mehta, J. S., et al. (2022) · Frontiers in Genetics · doi:10.3389/fgene.2021.794805 · Cited by 58 · see citing works

AAV for Gene Therapy in Ocular Diseases: Progress and Prospects

He, X., Fu, Y., Ma, L., Yao, Y., Ge, S., Yang, Z. H., & Fan, X. (2023) · Research · doi:10.34133/research.0291 · Cited by 59 · see citing works

Gene therapy and the adeno‐associated virus in the treatment of genetic and acquired ophthalmic diseases in humans: Trials, future directions and safety considerations

Ramlogan‐Steel, C. A., Murali, A., Andrzejewski, S., Dhungel, B. P., Steel, J. C., & Layton, C. J. (2018) · Clinical and Experimental Ophthalmology · doi:10.1111/ceo.13416 · Cited by 59 · see citing works

Materials promoting viral gene delivery

Kaygisiz, K., & Synatschke, C. V. (2020) · Biomaterials Science · doi:10.1039/d0bm01367f · Cited by 56 · see citing works

Bioinspired Underwater Propulsors

Buren, T. W. V., Floryan, D., & Smits, A. J. (2020) · Cambridge University Press eBooks · doi:10.1017/9781139058995.006 · Cited by 57 · see citing works

Safety of Adeno-associated virus-based vector-mediated gene therapy—impact of vector dose

Maurya, S., Sarangi, P., & Jayandharan, G. R. (2022) · Cancer Gene Therapy · doi:10.1038/s41417-021-00413-6 · Cited by 55 · see citing works

Assessment of quality attributes for adeno‐associated viral vectors

Tustian, A. D., & Bak, H. (2021) · Biotechnology and Bioengineering · doi:10.1002/bit.27905 · Cited by 53 · see citing works

U7 snRNA: A tool for gene therapy

Gadgil, A., & Raczyńska, K. D. (2021) · The Journal of Gene Medicine · doi:10.1002/jgm.3321 · Cited by 48 · see citing works

Changing trends in the development of AAV-based gene therapies: a meta-analysis of past and present therapies

Burdett, T., & Nuseibeh, S. (2022) · Gene Therapy · doi:10.1038/s41434-022-00363-0 · Cited by 51 · see citing works

Progress and challenges of gene therapy for Pompe disease

Ronzitti, G., Collaud, F., Laforêt, P., & Mingozzi, F. (2019) · Annals of Translational Medicine · doi:10.21037/atm.2019.04.67 · Cited by 52 · see citing works

Bone-Targeting AAV-Mediated Gene Silencing in Osteoclasts for Osteoporosis Therapy

Yang, Y.-S., Xie, J., Chaugule, S. R., Wang, D., Kim, J., Kim, J., Tai, P. W. L., Seo, S. K., Gravallese, E. M., Gao, G., et al. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.04.010 · Cited by 51 · see citing works

Structural dynamics, phase behavior, and applications of polyelectrolyte complex micelles

Shah, S. A., & Leon, L. (2021) · Current Opinion in Colloid & Interface Science · doi:10.1016/j.cocis.2021.101424 · Cited by 51 · see citing works

Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors

Ferla, R., Alliegro, M., Dell’Anno, M., Nusco, E., Cullen, J. M., Smith, S. N., Wolfsberg, T. G., O’Donnell, P., Wang, P., Nguyen, A., et al. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.11.015 · Cited by 50 · see citing works

Droplet Digital PCR Detection of the Erythropoietin Transgene from Horse Plasma and Urine for Gene-Doping Control

Tozaki, T., Ohnuma, A., Takasu, M., Kikuchi, M., Kakoi, H., HIROTA, K.-I., Kusano, K., & NAGATA, S.-I. (2019) · Genes · doi:10.3390/genes10030243 · Cited by 50 · see citing works

Gene Therapy Developments for Pompe Disease

Unnisa, Z., Yoon, J. K., Schindler, J. W., Mason, C., & van Til, N. P. (2022) · Biomedicines · doi:10.3390/biomedicines10020302 · Cited by 52 · see citing works

Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside

Xu, L., Yao, S., Ding, Y. E., Xie, M., Feng, D., Sha, P., Tan, L., Bei, F., & Yao, Y. (2024) · Journal of Translational Medicine · doi:10.1186/s12967-024-05661-2 · Cited by 48 · see citing works

Single-Stranded DNA-Packaged Polyplex Micelle as Adeno-Associated-Virus-Inspired Compact Vector to Systemically Target Stroma-Rich Pancreatic Cancer

Tockary, T. A., Foo, W., Dirisala, A., Chen, Q., Uchida, S., Osawa, S., Mochida, Y., Liu, X., Kinoh, H., Cabral, H., et al. (2019) · ACS Nano · doi:10.1021/acsnano.9b04676 · Cited by 42 · see citing works

Suppression of heterotopic ossification in fibrodysplasia ossificans progressiva using AAV gene delivery

Yang, Y.-S., Kim, J., Xie, J., Chaugule, S. R., Lin, C., Ma, H., Hsiao, E. C., Hong, J., Chun, H., Shore, E. M., et al. (2022) · Nature Communications · doi:10.1038/s41467-022-33956-9 · Cited by 43 · see citing works

Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates

Ballon, D., Rosenberg, J. B., Fung, E. K., Nikolopoulou, A., Kothari, P. J., De, B. P., He, B., Chen, A., Heier, L., Sondhi, D., et al. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.116 · Cited by 37 · see citing works

Circulating neurofilament light chain as a promising biomarker of AAV-induced dorsal root ganglia toxicity in nonclinical toxicology species

Fader, K. A., Pardo, I. D. R., Kovi, R. C., Somps, C. J., Wang, H. H., Vaidya, V. S., Ramaiah, S. K., & Sirivelu, M. P. (2022) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2022.03.017 · Cited by 40 · see citing works

Gene Therapy for Mucopolysaccharidosis Type II—A Review of the Current Possibilities

Zapolnik, P., & Pyrkosz, A. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22115490 · Cited by 39 · see citing works

Baculovirus-Mediated miRNA Regulation to Suppress Hepatocellular Carcinoma Tumorigenicity and Metastasis

Chen, C.-L., Wu, J., Chen, G., Yuan, P.-H., Tseng, Y.-W., Li, K.-C., Hwang, S., & Hu, Y.-C. (2014) · Molecular Therapy · doi:10.1038/mt.2014.126 · Cited by 37 · see citing works

Monitoring cell-mediated immune responses in AAV gene therapy clinical trials using a validated IFN-γ ELISpot method

Patton, K. S., Harrison, M. T., Long, B. R., Lau, K., Holcomb, J., Owen, R. E., Kasprzyk, T., Janetzki, S., Zoog, S. J., & Vettermann, C. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.05.012 · Cited by 37 · see citing works

Gene therapy for genetic diseases: challenges and future directions

Beibei, Q. I. E., Tuo, J., Chen, F., Ding, H., & Lyu, L. (2025) · MedComm · doi:10.1002/mco2.70091 · Cited by 37 · see citing works

Gene therapy of yeast NDI1 on mitochondrial complex I dysfunction in rotenone-induced Parkinson’s disease models in vitro and vivo

Li, H., Sun, B., Huang, Y., Zhang, J., Xu, X., Shen, Y., Chen, Z., Yang, J., Shen, L., Hu, Y., et al. (2022) · Molecular Medicine · doi:10.1186/s10020-022-00456-x · Cited by 34 · see citing works

Gene targeting as a therapeutic avenue in diseases mediated by the complement alternative pathway

Dreismann, A. K., Hallam, T. M., Tam, L. C. S., Nguyen, C. V., Hughes, J. P., Ellis, S., & Harris, C. L. (2022) · Immunological Reviews · doi:10.1111/imr.13149 · Cited by 33 · see citing works

Viral Vector-Mediated Antisense Therapy for Genetic Diseases

Imbert, M., Dias-Florencio, G., & Goyenvalle, A. (2017) · Genes · doi:10.3390/genes8020051 · Cited by 32 · see citing works

Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?

Zhan, W., Muhuri, M., Tai, P. W. L., & Gao, G. (2021) · Frontiers in Immunology · doi:10.3389/fimmu.2021.673699 · Cited by 33 · see citing works

A Roadmap for the Success of Oncolytic Parvovirus-Based Anticancer Therapies

Hartley, A., Kavishwar, G., Salvato, I., & Marchini, A. (2020) · Annual Review of Virology · doi:10.1146/annurev-virology-012220-023606 · Cited by 32 · see citing works

Manipulation of mitochondrial genes and mtDNA heteroplasmy

Bacman, S. R., Gammage, P. A., Minczuk, M. A., & Moraes, C. T. (2020) · Methods in cell biology · doi:10.1016/bs.mcb.2019.12.004 · Cited by 30 · see citing works

Programmable Assembly of Adeno-Associated Virus–Antibody Composites for Receptor-Mediated Gene Delivery

Zdechlik, A. C., He, Y., Aird, E. J., Gordon, W. R., & Schmidt, D. (2019) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.9b00790 · Cited by 28 · see citing works

Relationship between neutralizing antibodies against adeno-associated virus in the vitreous and serum: effects on retinal gene therapy

Lee, S., Kang, I. K., Kim, J. H., Jung, B. K., Park, K., Chang, H., Lee, J. Y., & Lee, H. (2019) · Translational Vision Science &amp; Technology · doi:10.1167/tvst.8.2.14 · Cited by 27 · see citing works

Rapid, scalable, and low-cost purification of recombinant adeno-associated virus produced by baculovirus expression vector system

Buclez, P.-O., Leite, G. D. F., Relizani, K., Beley, C., Garcı́a, L., & Benchaouir, R. (2016) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2016.35 · Cited by 26 · see citing works

Inhibition of Hepatitis B Virus by AAV8-Derived CRISPR/SaCas9 Expressed From Liver-Specific Promoters

Yan, K., Feng, J., Liu, X., Wang, H., Li, Q., Li, J., Xu, T., Sajid, M., Ullah, H., Zhou, L., et al. (2021) · Frontiers in Microbiology · doi:10.3389/fmicb.2021.665184 · Cited by 26 · see citing works

Scientific and Regulatory Policy Committee Points to Consider: Nonclinical Research and Development of In Vivo Gene Therapy Products, Emphasizing Adeno-Associated Virus Vectors

Hutt, J. A., Assaf, B. T., Bolon, B. N., Cavagnaro, J. A., Galbreath, E. J., Grubor, B., Kattenhorn, L. M., Romeike, A., & Whiteley, L. O. (2021) · Toxicologic Pathology · doi:10.1177/01926233211041962 · Cited by 27 · see citing works

PCR-Based Analytical Methods for Quantification and Quality Control of Recombinant Adeno-Associated Viral Vector Preparations

Shmidt, A. A., & Egorova, T. V. (2021) · Pharmaceuticals · doi:10.3390/ph15010023 · Cited by 26 · see citing works

Genetic Therapy Approaches for Ornithine Transcarbamylase Deficiency

Yılmaz, B. Ş., & Gissen, P. (2023) · Biomedicines · doi:10.3390/biomedicines11082227 · Cited by 26 · see citing works

Systemic administration of AAV-Slc25a46 mitigates mitochondrial neuropathy in Slc25a46−/− mice

Yang, L., Slone, J. D., Li, Z., Lou, X., Hu, Y., Queme, L. F., Jankowski, M. P., & Huang, T. (2020) · Human Molecular Genetics · doi:10.1093/hmg/ddz277 · Cited by 26 · see citing works

Accurate Quantification of AAV Vector Genomes by Quantitative PCR

de la Cámara, C. M.-F., McClements, M. E., & MacLaren, R. E. (2021) · Genes · doi:10.3390/genes12040601 · Cited by 25 · see citing works

In vivo CAR cell therapy: from bench to bedside

Jia, X., Chen, Z., Su, L., Ren, A., & Mei, H. (2025) · Journal of Hematology & Oncology · doi:10.1186/s13045-025-01759-2 · Cited by 27 · see citing works

Seek and destroy: targeted adeno-associated viruses for gene delivery to hepatocellular carcinoma

Dhungel, B. P., Jayachandran, A., Layton, C. J., & Steel, J. C. (2017) · Drug Delivery · doi:10.1080/10717544.2016.1247926 · Cited by 24 · see citing works

Current AAV-mediated gene therapy in sensorineural hearing loss

Qi, J., Fu, X., Zhang, L., Tan, F., Li, N., Sun, Q., Hu, X., He, Z., Xia, M., & Chai, R. (2022) · Fundamental Research · doi:10.1016/j.fmre.2022.08.015 · Cited by 24 · see citing works

Precision Fluorescent Labeling of an Adeno‐Associated Virus Vector to Monitor the Viral Infection Pathway

Zhang, C., Zhou, X., Yao, T., Tian, Z., & Zhou, D. (2018) · Biotechnology Journal · doi:10.1002/biot.201700374 · Cited by 24 · see citing works

Tetramer-Based Enrichment of Preexisting Anti-AAV8 CD8+ T Cells in Human Donors Allows the Detection of a TEMRA Subpopulation

Vandamme, C., Xicluna, R., Hesnard, L., Devaux, M., Jaulin, N., Guilbaud, M., Le Duff, J., Couzinié, C., Moullier, P., Saulquin, X., et al. (2020) · Frontiers in Immunology · doi:10.3389/fimmu.2019.03110 · Cited by 23 · see citing works

Neonatal neuronal WWOX gene therapy rescues Wwox null phenotypes

Repudi, S., Kustanovich, I. M., Abu‐Swai, S., Stern, S., & Aqeilan, R. I. (2021) · EMBO Molecular Medicine · doi:10.15252/emmm.202114599 · Cited by 23 · see citing works

Prospects for gene replacement therapies in amyotrophic lateral sclerosis

Giovannelli, I., Higginbottom, A., Kirby, J., Azzouz, M., & Shaw, P. J. (2022) · Nature Reviews Neurology · doi:10.1038/s41582-022-00751-5 · Cited by 23 · see citing works

Characterization of Virus Particles and Submicron-Sized Particulate Impurities in Recombinant Adeno-Associated Virus Drug Product

Hiemenz, C., Pacios-Michelena, A., Helbig, C., Vezočnik, V., Strebl, M., Nikels, F., Hawe, A., Garidel, P., & Menzen, T. (2023) · Journal of Pharmaceutical Sciences · doi:10.1016/j.xphs.2023.05.009 · Cited by 22 · see citing works

Cellular unfolded protein response against viruses used in gene therapy

Sen, D., Balakrishnan, B., & Jayandharan, G. R. (2014) · Frontiers in Microbiology · doi:10.3389/fmicb.2014.00250 · Cited by 23 · see citing works

Superior human hepatocyte transduction with adeno-associated virus vector serotype 7

Shao, W., Pei, X., Cui, C., Askew, C., Dobbins, A., Chen, X., Abajas, Y. L., Gerber, D. A., Samulski, R. J., Nichols, T. C., et al. (2019) · Gene Therapy · doi:10.1038/s41434-019-0104-5 · Cited by 22 · see citing works

A Small Virus to Deliver Small Antibodies: New Targeted Therapies Based on AAV Delivery of Nanobodies

Silva-Pilipich, N., Smerdou, C., & Vanrell, L. (2021) · Microorganisms · doi:10.3390/microorganisms9091956 · Cited by 21 · see citing works

A robust and efficient alluvial filtration method for the clarification of adeno-associated viruses from crude cell lysates

Meierrieks, F., Pickl, A., & Wolff, M. W. (2023) · Journal of Biotechnology · doi:10.1016/j.jbiotec.2023.03.010 · Cited by 23 · see citing works

Endocytosis in gene therapy with non-viral vectors

de Garibay, A. P. R. (2016) · Wiener Medizinische Wochenschrift · doi:10.1007/s10354-016-0450-5 · Cited by 21 · see citing works

Inducible HEK293 AAV packaging cell lines expressing Rep proteins

Jalšić, L., Lytvyn, V., Elahi, S. M., Hrapovic, S., Nassoury, N., Chahal, P. S., Gaillet, B., & Gilbert, R. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.07.002 · Cited by 20 · see citing works

Advances in Recombinant Adeno-Associated Virus Vectors for Neurodegenerative Diseases

Li, L., Vasan, L., Kartono, B., Clifford, K. P., Attarpour, A., Sharma, R., Mandrozos, M., Kim, A., Zhao, W., Belotserkovsky, A., et al. (2023) · Biomedicines · doi:10.3390/biomedicines11102725 · Cited by 19 · see citing works

Correction of a CADASIL point mutation using adenine base editors in hiPSCs and blood vessel organoids

Wang, J., Zhang, L., Wu, G., Wu, J., Zhou, X., Chen, X., Liu, Q., Jiao, Y., Liu, Q., Liang, P., et al. (2023) · Journal of genetics and genomics/Journal of Genetics and Genomics · doi:10.1016/j.jgg.2023.04.013 · Cited by 19 · see citing works

Current and Future Treatment of Mucopolysaccharidosis (MPS) Type II: Is Brain-Targeted Stem Cell Gene Therapy the Solution for This Devastating Disorder?

Horgan, C., Jones, S. A., Bigger, B., & Wynn, R. F. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms23094854 · Cited by 18 · see citing works

Translatability barriers between preclinical and clinical trials of AAV gene therapy in inherited retinal diseases

Shamshad, A., Kang, C., Jenny, L. A., Persad‐Paisley, E. M., & Tsang, S. H. (2023) · Vision Research · doi:10.1016/j.visres.2023.108258 · Cited by 16 · see citing works

Distinct chemical degradation pathways of AAV1 and AAV8 under thermal stress conditions revealed by analytical anion exchange chromatography and LC-MS-based peptide mapping

Xing, T., Li, S., Tang, S., Huang, Y., Liu, G., Yan, Y., Liu, D., Wang, S., Zhi, L., Shameem, M., et al. (2024) · Journal of Pharmaceutical and Biomedical Analysis · doi:10.1016/j.jpba.2024.116452 · Cited by 16 · see citing works

Adeno-associated virus vector system controlling capsid expression improves viral quantity and quality

Ohba, K., Sehara, Y., Enoki, T., Mineno, J., Ozawa, K., & Mizukami, H. (2023) · iScience · doi:10.1016/j.isci.2023.106487 · Cited by 16 · see citing works

Biophysical characterization of adeno-associated virus capsid through the viral transduction life cycle

Xiang, Y. S., & Hao, G. G. (2023) · Journal of Genetic Engineering and Biotechnology · doi:10.1186/s43141-023-00518-5 · Cited by 17 · see citing works

Selection of appropriate non-clinical animal models to ensure translatability of novel AAV-gene therapies to the clinic

Singh, M., Brööks, A. G., Toofan, P., & McLuckie, K. (2023) · Gene Therapy · doi:10.1038/s41434-023-00417-x · Cited by 15 · see citing works

Context-Specific Function of the Engineered Peptide Domain of PHP.B

Martino, R. A., Fluck, E. C., Murphy, J., Wang, Q., Hoff, H. B., Pumroy, R. A., Lee, C. Y., Sims, J. J., Roy, S., Moiseenkova‐Bell, V. Y., et al. (2021) · Journal of Virology · doi:10.1128/jvi.01164-21 · Cited by 16 · see citing works

Prevalence of Anti-Adeno-Associated Virus Serotype 9 Antibodies in Adult Patients with Spinal Muscular Atrophy

Stolte, B., Schreiber-Katz, O., Günther, R., Wurster, C. D., Petri, S., Osmanovic, A., Freigang, M., Uzelac, Z., Leo, M., von Velsen, O., et al. (2022) · Human Gene Therapy · doi:10.1089/hum.2022.054 · Cited by 15 · see citing works

Emerging Technologies Tackling Adeno-Associated Viruses (AAV) Immunogenicity in Gene Therapy Applications

Egorova, T. V., Starikova, A. V., & Polikarpova, A. V. (2025) · Pharmaceutics · doi:10.3390/pharmaceutics17111492 · Cited by 17 · see citing works

A Novel and Simplified Anion Exchange Flow‐Through Polishing Approach for the Separation of Full From Empty Adeno‐Associated Virus Capsids

Meierrieks, F., Weltken, A., Pflanz, K. B., Pickl, A., Graf, B., & Wolff, M. W. (2024) · Biotechnology Journal · doi:10.1002/biot.202400430 · Cited by 15 · see citing works

Progress in AAV-Mediated In Vivo Gene Therapy and Its Applications in Central Nervous System Diseases

Wang, S., & Xiao, L. (2025) · International Journal of Molecular Sciences · doi:10.3390/ijms26052213 · Cited by 15 · see citing works

Optimized Subretinal Injection Technique for Gene Therapy Approaches

Mühlfriedel, R., Michalakis, S., Garrido, M. G., Sothilingam, V., Schön, C., Biel, M., & Seeliger, M. W. (2019) · Methods in Molecular Biology · doi:10.1007/978-1-4939-8669-9_26 · Cited by 14 · see citing works

Emerging therapeutic potential of adeno-associated virus-mediated gene therapy in liver fibrosis

Bu, F., Jia, P., Zhu, Y. Y., Yang, Y., Meng, H., Bi, Y., Huang, C., & Li, J. (2022) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2022.06.009 · Cited by 13 · see citing works

Adeno-associated Virus Vectors in Gene Therapy

Mary, B., Khan, N., Arumugam, S., Saxena, H., Kumar, M., Manimaran, P., Chattopadhyay, S., & Jayandharan, G. R. (2018) · Springer eBooks · doi:10.1007/978-981-13-0481-1_2 · Cited by 124 · see citing works

Understanding and Tackling Immune Responses to Adeno-Associated Viral Vectors

Verdera, H. C., Unzu, C., Valeri, E., Adriouch, S., González‐Aseguinolaza, G., Mingozzi, F., & Kajaste‐Rudnitski, A. (2023) · Human Gene Therapy · doi:10.1089/hum.2023.119 · Cited by 40 · see citing works

Improvements and Applications of Adeno-associated Virus Vectors for Gene Therapy

Ding, W. (2009) · Journal of Capital Medical University

Developing Immunologically Inert Adeno-Associated Virus (AAV) Vectors for Gene Therapy: Possibilities and Limitations

Selot, R., Hareendran, S., & Jayandharan, G. R. (2014) · Current Pharmaceutical Biotechnology · doi:10.2174/1389201015666140327141710 · Cited by 31 · see citing works

Recent developments in adeno‐associated virus vector technology

Büning, H., Perabò, L., Coutelle, O., Quadt‐Humme, S., & Hallek, M. J. (2008) · The Journal of Gene Medicine · doi:10.1002/jgm.1205 · Cited by 189 · see citing works