Tailoring the AAV vector capsid for 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

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

Membrane-Associated Heparan Sulfate Proteoglycan Is a Receptor for Adeno-Associated Virus Type 2 Virions

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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

Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson’s disease: an open label, phase I trial

Kaplitt, M. G., Feigin, A., Tang, C., Fitzsimons, H. L., Mattis, P., Lawlor, P. A., Bland, R. J., Young, D., Strybing, K., Eidelberg, D., et al. (2007) · The Lancet · doi:10.1016/s0140-6736(07)60982-9 · Cited by 1,067 · see citing works

Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector

Kay, M. A., Manno, C. S., Ragni, M. V., Larson, P. J., Couto, L. B., McClelland, A., Glader, B. E., Chew, A. K., Tai, S. J., Herzog, R. W., et al. (2000) · Nature Genetics · doi:10.1038/73464 · Cited by 1,019 · see citing works

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

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

Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses

Calcedo, R., Vandenberghe, L. H., Gao, G., Lin, J., & Wilson, J. M. (2009) · The Journal of Infectious Diseases · doi:10.1086/595830 · Cited by 808 · 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

Cd8(+) T-Cell Responses to Adeno-Associated Virus Capsid in Humans

Mingozzi, F., Maus, M. V., Hui, D. J., Sabatino, D. E., Murphy, S. L., Rasko, J. E. J., Ragni, M. V., Manno, C. S., Sommer, J., Jiang, H., et al. (2007) · Nature Medicine · doi:10.1038/nm1549 · Cited by 748 · see citing works

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

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

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

Grimm, D., Lee, J. S., Wang, L., Desai, T., Akache, B., Storm, T. A., & Kay, M. A. (2008) · Journal of Virology · doi:10.1128/jvi.00254-08 · Cited by 696 · 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

Production and characterization of adeno-associated viral vectors

Grieger, J. C., Choi, V. W., & Samulski, R. J. (2006) · Nature Protocols · doi:10.1038/nprot.2006.207 · Cited by 567 · see citing works

Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors

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Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector

Herzog, R. W., Yang, E. Y., Couto, L. B., Hagstrom, J. N., Elwell, D., Fields, P. A., Burton, M., Bellinger, D. A., Read, M. S., Brinkhous, K. M., et al. (1999) · Nature Medicine · doi:10.1038/4743 · Cited by 554 · see citing works

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

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

New recombinant serotypes of AAV vectors

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

Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus

Herzog, R. W., Hagstrom, J. N., Kung, S.-H., Tai, S. J., Wilson, J. M., Fisher, K. J., & High, K. A. (1997) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.94.11.5804 · Cited by 474 · see citing works

Cloning of adeno-associated virus into pBR322: Rescue of intact virus from the recombinant plasmid in human cells

Samulski, R. J., Berns, K. I., Tan, M., & Muzyczka, N. (1982) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.79.6.2077 · Cited by 466 · see citing works

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

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

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

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

Adeno-Associated Virus Serotype 4 (AAV4) and AAV5 Both Require Sialic Acid Binding for Hemagglutination and Efficient Transduction but Differ in Sialic Acid Linkage Specificity

Kaludov, N. K., Brown, K. E., Walters, R. W., Zabner, J., & Chiorini, J. A. (2001) · Journal of Virology · doi:10.1128/jvi.75.15.6884-6893.2001 · Cited by 428 · 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

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

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

Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2

Girod, A., Ried, M., Wobus, C., Lahm, H., Leike, K., Kleinschmidt, J., Deléage, G., & Hallek, M. (1999) · Nature Medicine · doi:10.1038/12491 · Cited by 405 · see citing works

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA

Worgall, S., Sondhi, D., Hackett, N. R., Kosofsky, B., Kekatpure, M. V., Neyzi, N., Dyke, J. P., Ballon, D., Heier, L., Greenwald, B. M., et al. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.022 · Cited by 402 · see citing works

Differential activation of innate immune responses by adenovirus and adeno-associated virus vectors

Zaiss, A.-K., Liu, Q., Bowen, G. P., Wong, N. C. W., Bartlett, J. S., & Muruve, D. A. (2002) · Journal of Virology · doi:10.1128/jvi.76.9.4580-4590.2002 · Cited by 394 · see citing works

Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model

Auricchio, A. (2001) · Human Molecular Genetics · doi:10.1093/hmg/10.26.3075 · Cited by 394 · see citing works

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

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

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

Results from a phase I safety trial of <i>hAADC</i> gene therapy for Parkinson disease

Eberling, J. L., Jagust, W. J., Christine, C. W., Starr, P., Larson, P., Bankiewicz, K. S., & Aminoff, M. J. (2008) · Neurology · doi:10.1212/01.wnl.0000312381.29287.ff · Cited by 370 · 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

Rapid Uncoating of Vector Genomes Is the Key toEfficient Liver Transduction with Pseudotyped Adeno-Associated VirusVectors

Thomas, C. E., Storm, T. A., Huang, Z., & Kay, M. A. (2004) · Journal of Virology · doi:10.1128/jvi.78.6.3110-3122.2004 · Cited by 358 · see citing works

Identification of PDGFR as a receptor for AAV-5 transduction

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

From Virus Evolution to Vector Revolution: Use of Naturally Occurring Serotypes of Adeno-associated Virus (AAV) as Novel Vectors for Human Gene Therapy

Grimm, D., & Kay, M. A. (2003) · Current Gene Therapy · doi:10.2174/1566523034578285 · Cited by 349 · 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

Adeno-Associated Virus Type 5 (AAV5) but Not AAV2 Binds to the Apical Surfaces of Airway Epithelia and Facilitates Gene Transfer

Zabner, J., Seiler, M. P., Walters, R. H., Kotin, R., Fulgeras, W., Davidson, B., & Chiorini, J. (2000) · Journal of Virology · doi:10.1128/jvi.74.8.3852-3858.2000 · Cited by 329 · see citing works

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

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

Adeno-associated viruses undergo substantial evolution in primates during natural infections

Gao, G., Alvira, M. R., Somanathan, S., Lü, Y., Vandenberghe, L. H., Rux, J. J., Calcedo, R., Sanmiguel, J. C., Abbas, Z., & Wilson, J. M. (2003) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0937739100 · Cited by 323 · see citing works

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

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

Adeno-Associated Virus Type 6 (AAV6) Vectors Mediate Efficient Transduction of Airway Epithelial Cells in Mouse Lungs Compared to That of AAV2 Vectors

Halbert, C. L., Allen, J. M., & Miller, A. D. (2001) · Journal of Virology · doi:10.1128/jvi.75.14.6615-6624.2001 · Cited by 314 · see citing works

Isolation of Highly Infectious and Pure Adeno-Associated Virus Type 2 Vectors with a Single-Step Gravity-Flow Column

Auricchio, A., Hildinger, M., O'Connor, E., Gao, G., & Wilson, J. M. (2001) · Human Gene Therapy · doi:10.1089/104303401450988 · Cited by 296 · see citing works

Long-Term Clinical Improvement in MPTP-Lesioned Primates after Gene Therapy with AAV-hAADC

Bankiewicz, K. S., Forsayeth, J., Eberling, J. L., Sánchez‐Pernaute, R., Pivirotto, P., Bringas, J., Herscovitch, P., Carson, R. E., Eckelman, W. C., Reutter, B. W., et al. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.05.005 · Cited by 282 · see citing works

Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer

Rivera, V. M., Gao, G., Grant, R. L., Schnell, M. A., Zoltick, P. W., Rozamus, L. W., Clackson, T., & Wilson, J. M. (2004) · Blood · doi:10.1182/blood-2004-06-2501 · Cited by 272 · see citing works

Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates

Nathwani, A. C., Gray, J. T., McIntosh, J., Ng, C. Y., Zhou, J., Spence, Y., Cochrane, M., Gray, E., Tuddenham, E. G. D., & Davidoff, A. M. (2006) · Blood · doi:10.1182/blood-2006-03-010181 · Cited by 272 · see citing works

Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice

Scallan, C. D., Jiang, H., Liu, T., Patarroyo-White, S., Sommer, J. M., Zhou, S., Couto, L. B., & Pierce, G. F. (2006) · Blood · doi:10.1182/blood-2005-08-3229 · Cited by 271 · see citing works

A Phase II, Double-Blind, Randomized, Placebo-Controlled Clinical Trial of tgAAVCF Using Maxillary Sinus Delivery in Patients with Cystic Fibrosis with Antrostomies

Wagner, J. A., Nepomuceno, I. B., Messner, A. H., Moran, M. L., Batson, E. P., Dimiceli, S., Brown, B. W., Desch, J. K., Norbash, A., Conrad, C., et al. (2002) · Human Gene Therapy · doi:10.1089/104303402760128577 · Cited by 265 · see citing works

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

Li, W., Asokan, A., Wu, Z., Van Dyke, T., DiPrimio, N., Johnson, J. S., Govindaswamy, L., Agbandje-McKenna, M., Leichtle, S., Eugene Redmond Jr, D., et al. (2008) · Molecular Therapy · doi:10.1038/mt.2008.100 · Cited by 261 · 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

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

Single Amino Acid Changes Can Influence Titer, Heparin Binding, and Tissue Tropism in Different Adeno-Associated Virus Serotypes

Wu, Z., Asokan, A., Grieger, J. C., Lakshmanan Govindasamy, A., Agbandje‐McKenna, M., & Samulski, R. J. (2006) · Journal of Virology · doi:10.1128/jvi.01288-06 · Cited by 239 · see citing works

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

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

Characterization of Adeno-Associated Virus Genomes Isolated from Human Tissues

Schnepp, B. C., Jensen, R. L., Chen, C.-L., Johnson, P. R., & Clark, K. R. (2005) · Journal of Virology · doi:10.1128/jvi.79.23.14793-14803.2005 · Cited by 231 · see citing works

Design of a Phase 1/2 Trial of Intracoronary Administration of AAV1/SERCA2a in Patients With Heart Failure

Hajjar, R. J., Zsebo, K. M., Deckelbaum, L. I., Thompson, C., Rudy, J., Yaroshinsky, A., Ly, H. Q., Kawase, Y., Wagner, K., Borow, K., et al. (2008) · Journal of Cardiac Failure · doi:10.1016/j.cardfail.2008.02.005 · Cited by 220 · see citing works

Sustained AAV-mediated Dystrophin Expression in a Canine Model of Duchenne Muscular Dystrophy with a Brief Course of Immunosuppression

Wang, Z., Kuhr, C. S., Allen, J. M., Blankinship, M. J., Gregorevic, P., Chamberlain, J. S., Tapscott, S. J., & Storb, R. F. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300161 · Cited by 218 · see citing works

Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models

Davidoff, A. M., Gray, J. T., Ng, C. Y., Zhang, Y., Zhou, J., Spence, Y., Bakar, Y., & Nathwani, A. C. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2004.12.022 · Cited by 218 · see citing works

Immune Responses to Adeno-Associated Virus Vectors

Zaiss, A. K., & Muruve, D. A. (2005) · Current Gene Therapy · doi:10.2174/1566523054065039 · Cited by 214 · see citing works

Safety and Biological Efficacy of an Adeno‐Associated Virus Vector–Cystic Fibrosis Transmembrane Regulator (AAV‐CFTR) in the Cystic Fibrosis Maxillary Sinus

Wagner, J. A., Messner, A. H., Moran, M. L., Daifuku, R., Kouyama, K., Desch, J. K., Manley, S. A., Norbash, A., Conrad, C., Friborg, S., et al. (1999) · The Laryngoscope · doi:10.1097/00005537-199902000-00017 · Cited by 209 · see citing works

Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid

Vandenberghe, L. H., Wang, L., Somanathan, S., Zhi, Y., Figueredo, J., Calcedo, R., Sanmiguel, J., Desai, R. A., Chen, C. S., Johnston, J., et al. (2006) · Nature Medicine · doi:10.1038/nm1445 · Cited by 208 · see citing works

Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy

Grimm, D., Zhou, S., Nakai, H., Thomas, C. E., Storm, T. A., Fuess, S., Matsushita, T., Allen, J. M., Surosky, R. T., Lochrie, M. A., et al. (2003) · Blood · doi:10.1182/blood-2003-02-0495 · Cited by 208 · 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

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 · 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

Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung.

Conrad, C., Allen, S. S., Afione, S. A., Reynolds, T. C., Beck, S. E., Fee-Maki, M., Barraza-Ortiz, X., Adams, R. J., Askin, F. B., Carter, B. J., et al. (1996) · PubMed · Cited by 196 · see citing works

Subthalamic GAD gene transfer in Parkinson disease patients who are candidates for deep brain stimulation.

During, M. J., Kaplitt, M. G., Stern, M. B., et al. (2001) · PubMed · Cited by 181 · see citing works

Sustained Expression of Therapeutic Level of Factor IX in Hemophilia B Dogs by AAV-Mediated Gene Therapy in Liver

Wang, L., Nichols, T. C., Read, M. S., Bellinger, D. A., & Verma, I. M. (2000) · Molecular Therapy · doi:10.1006/mthe.2000.0031 · Cited by 176 · see citing works

Sustained correction of disease in naive and AAV2-pretreated hemophilia b dogs: AAV2/8-mediated, liver-directed gene therapy

Wang, L., Calcedo, R., Nichols, T. C., Bellinger, D. A., Dillow, A., Verma, I. M., & Wilson, J. M. (2005) · Blood · doi:10.1182/blood-2004-10-3867 · Cited by 173 · see citing works

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

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In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector

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Scalable Purification of Adeno-Associated Virus Type 2, 4, or 5 Using Ion-Exchange Chromatography

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Liver Transduction with Recombinant Adeno-Associated Virus Is Primarily Restricted by Capsid Serotype Not Vector Genotype

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Pre-existing AAV Capsid-specific CD8+ T Cells are Unable to Eliminate AAV-transduced Hepatocytes

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Structure of Adeno-Associated Virus Type 4

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Long-Term Efficacy of Adeno-Associated Virus Serotypes 8 and 9 in Hemophilia A Dogs and Mice

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Local delivery of a recombinant adenoassociated vector containing a tumour necrosis factor α antagonist gene in inflammatory arthritis: a phase 1 dose-escalation safety and tolerability study

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A Limited Number of Transducible Hepatocytes Restricts a Wide-Range Linear Vector Dose Response in Recombinant Adeno-Associated Virus-Mediated Liver Transduction

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Cloning of an Avian Adeno-Associated Virus (AAAV) and Generation of Recombinant AAAV Particles

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Immune Responses to Recombinant Adeno-Associated Virus Vectors: Putting Preclinical Findings into Perspective

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Therapeutic potential of CERE-110 (AAV2-NGF): Targeted, stable, and sustained NGF delivery and trophic activity on rodent basal forebrain cholinergic neurons

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AAV Vector-mediated Reversal of Hypoglycemia in Canine and Murine Glycogen Storage Disease Type Ia

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Adeno-Associated Virus Type 2 (AAV2) Capsid-Specific Cytotoxic T Lymphocytes Eliminate Only Vector-Transduced Cells Coexpressing the AAV2 Capsid In Vivo

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Deposition and Expression of Aerosolized rAAV Vectors in the Lungs of Rhesus Macaques

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Cloning and Characterization of a Bovine Adeno-Associated Virus

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Correction of Feline Lipoprotein Lipase Deficiency with Adeno-Associated Virus Serotype 1-Mediated Gene Transfer of the Lipoprotein Lipase S447X Beneficial Mutation

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Percutaneous Transendocardial Delivery of Self-complementary Adeno-associated Virus 6 Achieves Global Cardiac Gene Transfer in Canines

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Subretinal Delivery of Recombinant AAV Serotype 8 Vector in Dogs Results in Gene Transfer to Neurons in the Brain

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Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints

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AAV as An Immunogen

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Gene Therapy for Lipoprotein Lipase Deficiency: Working Toward Clinical Application

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Identification and Characterization of Novel Adeno-Associated Virus Isolates in ATCC Virus Stocks

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Impact of Preexisting Vector Immunity on the Efficacy of Adeno-Associated Virus-Based HIV-1 Gag Vaccines

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Preclinical Characterization of A Recombinant Adeno-Associated Virus Type 1-Pseudotyped Vector Demonstrates Dose-Dependent Injection Site Inflammation And Dissemination of Vector Genomes to Distant Sites

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995. Effect of Viral Dose on Neutralizing Antibody Response and Transgene Expression after AAV1 Vector Re-Administration in Mice

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Phase I Trial of Leber Congenital Amaurosis due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results

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Addition of Six-His-Tagged Peptide to the C Terminus of Adeno-Associated Virus VP3 Does Not Affect Viral Tropism or Production

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Efficient AAV1–AAV2 Hybrid Vector for Gene Therapy of Hemophilia

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Gene therapeutic prospects in early onset of severe retinal dystrophy: restoration of vision in RPE65 Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium.

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Artificial Evolution with Adeno-Associated Viral Libraries

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Gene therapy advances: A meta-analysis of AAV usage in clinical settings

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Novel adeno-associated viral vectors for retinal gene therapy.

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Convolutions are competitive with transformers for protein sequence pretraining

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Molecular Therapies for Inherited Retinal Diseases—Current Standing, Opportunities and Challenges

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Adeno-associated Virus: A Key to the Human Genome?

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Synthetic Adeno-Associated Viral Vector Efficiently Targets Mouse and Nonhuman Primate Retina <i>In Vivo</i>

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Gene Therapy Using a <i>miniCEP290</i> Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis

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Comparison of adeno-associated virus serotypes and delivery methods for cardiac gene transfer

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Impact of Preexisting Vector Immunity on the Efficacy of Adeno-Associated Virus-Based HIV-1 Gag Vaccines

Lin, J., Calcedo, R., Vandenberghe, L. H., Figueredo, J. M., & Wilson, J. M. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.033 · Cited by 53 · see citing works

AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence

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Genome-wide RNAi screening identifies host restriction factors critical for in vivo AAV transduction

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Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUgt1a1 enzyme in a mouse model of crigler–najjar syndrome type I

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The Ocular Gene Delivery Landscape

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Effects of immunosuppression on circulating adeno-associated virus capsid-specific T cells in humans

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Chemical Modifications of the Capsid for Redirecting and Improving the Efficacy of Adeno-Associated Virus Vectors

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Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties

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Characterizing Adeno-Associated Virus Capsids with both Denaturing and Intact Analysis MethodsPreprint

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Capsid Antibodies to Different Adeno-Associated Virus Serotypes Bind Common Regions

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782. Adeno-Associated Virus Serotype-9 (AAV-9) Mediated Gene Transfer in the Mouse Retina

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