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

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

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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

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

Russell, S., Bennett, J., Wellman, J. A., Chung, D. C., Yu, Z.-F., Tillman, A., Wittes, J., Pappas, J., Elci, O., McCague, S., et al. (2017) · The Lancet · doi:10.1016/s0140-6736(17)31868-8 · Cited by 1,952 · 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

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

Production of High-Titer Recombinant Adeno-Associated Virus Vectors in the Absence of Helper Adenovirus

Xiao, X., Li, J., & Samulski, R. J. (1998) · Journal of Virology · doi:10.1128/jvi.72.3.2224-2232.1998 · Cited by 1,381 · see citing works

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

Nathwani, A. C., Reiss, U. M., Tuddenham, E. G. D., Rosales, C., Chowdary, P., McIntosh, J., Della Peruta, M., Lheriteau, E., Patel, N., Raj, D., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1407309 · Cited by 1,273 · 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

Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain

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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

Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors

Boutin, S., Monteilhet, V., Veron, P., Leborgne, C., Benveniste, O., Montus, M. F., & Masurier, C. (2010) · Human Gene Therapy · doi:10.1089/hum.2009.182 · Cited by 970 · see citing works

Treatment of Leber Congenital Amaurosis Due to <i>RPE65</i> Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

Hauswirth, W. W., Aleman, T. S., Kaushal, S., Cideciyan, A. V., Schwartz, S. B., Wang, L., Conlon, T. J., Boye, S. L., Flotte, T. R., Byrne, B. J., et al. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.107 · Cited by 954 · see citing works

Site-specific integration by adeno-associated virus.

Kotin, R. M., Siniscalco, M., Samulski, R. J., Zhu, X. D., Hunter, L., Laughlin, C. A., McLaughlin, S., Muzyczka, N., Rocchi, M., & Berns, K. I. (1990) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.87.6.2211 · Cited by 906 · see citing works

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

Hinderer, C., Katz, N., Buza, E. L., Dyer, C., Goode, T., Bell, P., Richman, L. K., & Wilson, J. M. (2018) · Human Gene Therapy · doi:10.1089/hum.2018.015 · Cited by 814 · see citing works

Site-specific integration by adeno-associated virus.

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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

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

Manno, C. S., Chew, A. J., Hutchison, S., Larson, P. J., Herzog, R. W., Arruda, V. R., Tai, S. J., Ragni, M. V., Thompson, A., Ozelo, M., et al. (2003) · Blood · doi:10.1182/blood-2002-10-3296 · Cited by 749 · 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

Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression

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Nucleotide sequence and organization of the adeno-associated virus 2 genome

Srivastava, A., Lusby, E. W., & Berns, K. I. (1983) · Journal of Virology · doi:10.1128/jvi.45.2.555-564.1983 · Cited by 721 · see citing works

Novel Tools for Production and Purification of Recombinant Adenoassociated Virus Vectors

Grimm, D., Kern, A., Rittner, K., & Kleinschmidt, J. A. (1998) · Human Gene Therapy · doi:10.1089/hum.1998.9.18-2745 · Cited by 700 · see citing works

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

Cideciyan, A. V., Alemán, T. S., Boye, S. L., Schwartz, S., Kaushal, S., Román, A. J., Pang, J., Sumaroka, A., Windsor, E. A. M., Wilson, J. M., et al. (2008) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0807027105 · Cited by 674 · see citing works

Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein

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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

Quantitative analysis of the packaging capacity of recombinant adeno-associated virus

Dong, J.-Y., Fan, P.-D., & Frizzell, R. A. (1996) · Human Gene Therapy · doi:10.1089/hum.1996.7.17-2101 · Cited by 583 · see citing works

Characterization of a preferred site on human chromosome 19q for integration of adeno‐associated virus DNA by non‐homologous recombination.

Kotin, R. M., Linden, R. M., & Berns, K. I. (1992) · The EMBO Journal · doi:10.1002/j.1460-2075.1992.tb05614.x · Cited by 513 · see citing works

Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector.

Flotte, T. R., Afione, S. A., Conrad, C., McGrath, S. A., Solow, R., Oka, H., Zeitlin, P. L., Guggino, W. B., & Carter, B. J. (1993) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.90.22.10613 · Cited by 490 · see citing works

Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas

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

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

Insect Cells as a Factory to Produce Adeno-Associated Virus Type 2 Vectors

Urabe, M., Ding, C., & Kotin, R. M. (2002) · Human Gene Therapy · doi:10.1089/10430340260355347 · Cited by 457 · see citing works

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

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

Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector

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A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication

Samulski, R. J., Chang, L., & Shenk, T. E. (1987) · Journal of Virology · doi:10.1128/jvi.61.10.3096-3101.1987 · Cited by 449 · see citing works

Safety and tolerability of putaminal <i>AADC</i> gene therapy for Parkinson disease

Christine, C. W., Starr, P. A., Larson, P. S., Eberling, J. L., Jagust, W. J., Hawkins, R. A., VanBrocklin, H. F., Wright, J. F., Bankiewicz, K. S., & Aminoff, M. J. (2009) · Neurology · doi:10.1212/wnl.0b013e3181c29356 · Cited by 445 · see citing works

Defective regulation of outwardly rectifying Cl− channels by protein kinase A corrected by insertion of CFTR

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The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice

Hordeaux, J., Wang, Q., Katz, N., Buza, E. L., Bell, P., & Wilson, J. M. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.01.018 · Cited by 426 · see citing works

Packaging Capacity of Adeno-Associated Virus Serotypes: Impact of Larger Genomes on Infectivity and Postentry Steps

Grieger, J. C., & Samulski, R. J. (2005) · Journal of Virology · doi:10.1128/jvi.79.15.9933-9944.2005 · Cited by 419 · see citing works

Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain

Cearley, C. N., & Wolfe, J. H. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2005.11.015 · Cited by 409 · 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

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

Gaudet, D., Méthot, J., Déry, S., Brisson, D., Essiembre, C., Tremblay, G., Tremblay, K., de Wal, J., Twisk, J., van den Bulk, N., et al. (2013) · Gene Therapy · doi:10.1038/gt.2012.43 · Cited by 404 · 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

Mutational Analysis of the Adeno-Associated Virus Type 2 (AAV2) Capsid Gene and Construction of AAV2 Vectors with Altered Tropism

Wu, P., Xiao, W. S., Conlon, T. J., Hughes, J. A., Agbandje‐McKenna, M., Ferkol, T. W., Flotte, T. R., & Muzyczka, N. (2000) · Journal of Virology · doi:10.1128/jvi.74.18.8635-8647.2000 · Cited by 390 · see citing works

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

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

A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson’s disease

Muramatsu, S.-I., Fujimoto, K.-I., Kato, S., Mizukami, H., Asari, S., Ikeguchi, K., Kawakami, T., Urabe, M., Kume, A., Sato, T., et al. (2010) · Molecular Therapy · doi:10.1038/mt.2010.135 · Cited by 344 · see citing works

Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6

Grimm, D., Kay, M. A., & Kleinschmidt, J. A. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(03)00095-9 · Cited by 344 · see citing works

Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter.

Flotte, T. R., Afione, S. A., Solow, R., Drumm, M. L., Markakis, D., Guggino, W. B., Zeitlin, P. L., & Carter, B. J. (1993) · Journal of Biological Chemistry · doi:10.1016/s0021-9258(18)53762-5 · Cited by 342 · see citing works

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α ₁ -Antitrypsin: Interim Results

Flotte, T. R., Trapnell, B. C., Humphries, M., Carey, B., Calcedo, R., Rouhani, F., Campbell-Thompson, M., Yachnis, A. T., Sandhaus, R. A., McElvaney, N. G., et al. (2011) · Human Gene Therapy · doi:10.1089/hum.2011.053 · Cited by 335 · see citing works

The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice

Zhu, J., Huang, X., & Yang, Y. (2009) · Journal of Clinical Investigation · doi:10.1172/jci37607 · Cited by 330 · see citing works

Mapping and direct visualization of a region-specific viral DNA integration site on chromosome 19q13-qter

Kotin, R. M., Menninger, J. C., Ward, D. C., & Berns, K. I. (1991) · Genomics · doi:10.1016/0888-7543(91)90470-y · Cited by 329 · see citing works

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

Brantly, M. L., Chulay, J. D., Wang, L., Mueller, C., Humphries, M., Spencer, L. T., Rouhani, F., Conlon, T. J., Calcedo, R., Betts, M. R., et al. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0904514106 · Cited by 327 · see citing works

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

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

Long-term follow-up after gene therapy for canavan disease

Leone, P., Shera, D., McPhee, S. W. J., Francis, J. S., Kolodny, E. H., Bilaniuk, L. T., Wang, D.-J., Assadi, M., Goldfarb, O., Goldman, H. W., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3003454 · Cited by 307 · see citing works

Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors

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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

Rescue of adeno-associated virus from recombinant plasmids: Gene correction within the terminal repeats of AAV

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Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice

Allocca, M., Doria, M., Petrillo, M., Colella, P., Garcia-Hoyos, M., Gibbs, D., Kim, S. R., Maguire, A., Rex, T. S., Di Vicino, U., et al. (2008) · Journal of Clinical Investigation · doi:10.1172/jci34316 · Cited by 292 · see citing works

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

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

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

McIntosh, J., Lenting, P. J., Rosales, C., Lee, D., Rabbanian, S., Raj, D., Patel, N., Tuddenham, E. G. D., Christophe, O. D., McVey, J. H., et al. (2013) · Blood · doi:10.1182/blood-2012-10-462200 · Cited by 278 · 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

Cloning of infectious adeno-associated virus genomes in bacterial plasmids

Laughlin, C. A., Tratschin, J.-D., Coon, H., & Carter, B. J. (1983) · Gene · doi:10.1016/0378-1119(83)90217-2 · Cited by 266 · 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

A compact, high-accuracy Cas9 with a dinucleotide PAM for in vivo genome editing

Edraki, A., Mir, A., Ibraheim, R., Gainetdinov, I., Yoon, Y., Song, C.-Q., Cao, Y., Gallant, J., Xue, W., Rivera-Pérez, J. A., et al. (2019) · Molecular Cell · doi:10.1016/j.molcel.2018.12.003 · Cited by 265 · see citing works

Expanding AAV Packaging Capacity with Trans-splicing or Overlapping Vectors: A Quantitative Comparison

Duan, D., Yue, Y., & Engelhardt, J. F. (2001) · Molecular Therapy · doi:10.1006/mthe.2001.0456 · Cited by 262 · see citing works

Intracerebral Administration of Adeno-Associated Viral Vector Serotype rh.10 Carrying Human <i>SGSH</i> and <i>SUMF1</i> cDNAs in Children with Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/II Trial

Tardieu, M., Zérah, M., Husson, B., de Bournonville, S., Deiva, K., Adamsbaum, C., Vincent, F., Hocquemiller, M., Broissand, C., Furlan, V., et al. (2014) · Human Gene Therapy · doi:10.1089/hum.2013.238 · Cited by 261 · 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

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

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

Cell Lines for the Production of Recombinant Adeno-Associated Virus

Clark, K. R., Voulgaropoulou, F., Fraley, D. M., & Johnson, P. R. (1995) · Human Gene Therapy · doi:10.1089/hum.1995.6.10-1329 · Cited by 257 · see citing works

Gene Therapy of Canavan Disease: AAV-2 Vector for Neurosurgical Delivery of Aspartoacylase Gene ( <i>ASPA</i> ) to the Human Brain

Janson, C., McPhee, S., Bilaniuk, L., Haselgrove, J., Testaiuti, M., Freese, A., Wang, D.-J., Shera, D., Hurh, P., Rupin, J., et al. (2002) · Human Gene Therapy · doi:10.1089/104303402760128612 · Cited by 255 · see citing works

Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver

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The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9–dependent innate immune responses in the liver

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Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity

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Customized blood-brain barrier shuttle peptide to increase AAV9 vector crossing the BBB and augment transduction in the brain

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Multiplexing AAV Serotype-Specific Neutralizing Antibodies in Preclinical Animal Models and Humans

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Gene Therapy for Cystic Fibrosis Paved the Way for the Use of Adeno-Associated Virus in Gene Therapy

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