Immune Responses to Muscle-Directed Adeno-Associated Viral Gene Transfer in Clinical Studies

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

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

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

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

Immune Responses to Viral Gene Therapy Vectors

Shirley, J. L., de Jong, Y. P., Terhorst, C., & Herzog, R. W. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.01.001 · Cited by 788 · 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

Current clinical applications of in vivo gene therapy with AAVs

Mendell, J. R., Al-Zaidy, S. A., Rodino-Klapac, L. R., Goodspeed, K., Gray, S. J., Kay, C. N., Boye, S. L., Boye, S. E., George, L. A., Salabarria, S., et al. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2020.12.007 · Cited by 743 · see citing works

Dystrophin Immunity in Duchenne's Muscular Dystrophy

Mendell, J. R., Campbell, K., Rodino-Klapac, L., Sahenk, Z., Shilling, C., Lewis, S., Bowles, D., Gray, S., Li, C., Galloway, G., et al. (2010) · New England Journal of Medicine · doi:10.1056/nejmoa1000228 · Cited by 655 · 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

Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species

Tabebordbar, M., Lagerborg, K. A., Stanton, A. C., King, E. M., Ye, S. H., Tellez, L., Krunnfusz, A., Tavakoli, S., Widrick, J. J., Messemer, K. A., et al. (2021) · Cell · doi:10.1016/j.cell.2021.08.028 · Cited by 517 · see citing works

IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

Leborgne, C., Barbon, E., Alexander, J. M., Hanby, H., Delignat, S., Cohen, D. M., Collaud, F., Muraleetharan, S., Lupo, D., Silverberg, J., et al. (2020) · Nature Medicine · doi:10.1038/s41591-020-0911-7 · Cited by 336 · 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

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

Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy

Chand, D., Mohr, F., McMillan, H., Tukov, F. F., Montgomery, K., Kleyn, A., Sun, R., Tauscher-Wisniewski, S., Kaufmann, P., & Kullak-Ublick, G. (2021) · Journal of Hepatology · doi:10.1016/j.jhep.2020.11.001 · Cited by 306 · see citing works

AAV-1–mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

Mingozzi, F., Meulenberg, J. J., Hui, D. J., Basner-Tschakarjan, E., Hasbrouck, N. C., Edmonson, S. A., Hutnick, N. A., Betts, M. R., Kastelein, J. J., Stroes, E. S., et al. (2009) · Blood · doi:10.1182/blood-2008-07-167510 · Cited by 295 · see citing works

Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants

Weinmann, J., Weis, S., Sippel, J., Tulalamba, W., Remes, A., El Andari, J., Herrmann, A.-K., Pham, Q. H., Borowski, C., Hille, S., et al. (2020) · Nature Communications · doi:10.1038/s41467-020-19230-w · Cited by 259 · see citing works

CpG-depleted adeno-associated virus vectors evade immune detection

Faust, S. M., Bell, P., Cutler, B. J., Ashley, S. N., Zhu, Y., Rabinowitz, J. E., & Wilson, J. M. (2013) · Journal of Clinical Investigation · doi:10.1172/jci68205 · Cited by 254 · see citing works

Sustained alpha‐sarcoglycan gene expression after gene transfer in limb‐girdle muscular dystrophy, type 2D

Mendell, J. R., Rodino‐Klapac, L. R., Rosales, X. Q., Coley, B. D., Galloway, G., Lewis, S., Malik, V., Shilling, C., Byrne, B. J., Conlon, T., et al. (2010) · Annals of Neurology · doi:10.1002/ana.22251 · Cited by 248 · see citing works

A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

Mendell, J. R., Sahenk, Z., Malik, V., Gomez, A. M., Flanigan, K. M., Lowes, L. P., Alfano, L. N., Berry, K., Meadows, E., Lewis, S., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.200 · Cited by 246 · see citing works

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

Martino, A. T., Suzuki, M., Markusic, D. M., Zolotukhin, I., Ryals, R. C., Moghimi, B., Ertl, H. C. J., Muruve, D. A., Lee, B., & Herzog, R. W. (2011) · Blood · doi:10.1182/blood-2010-10-314518 · Cited by 246 · see citing works

Limb‐girdle muscular dystrophy type 2D gene therapy restores α‐sarcoglycan and associated proteins

Mendell, J. R., Rodino‐Klapac, L. R., Rosales, X. Q., Kota, J., Coley, B. D., Galloway, G. M., Craenen, J. M., Lewis, S., Malik, V., Shilling, C. J., et al. (2009) · Annals of Neurology · doi:10.1002/ana.21732 · Cited by 214 · see citing works

BAX 335 hemophilia b gene therapy clinical trial results: Potential impact of CpG sequences on gene expression

Konkle, B. A., Walsh, C. E., Escobar, M. A., Josephson, N. C., Young, G., von Drygalski, A., McPhee, S. W. J., Samulski, R. J., Bilic, I., de la Rosa, M., et al. (2021) · Blood · doi:10.1182/blood.2019004625 · Cited by 189 · see citing works

Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Serotype 2 α 1-Antitrypsin (AAT) Vector in AAT-Deficient Adults

Brantly, M., Spencer, L. T., Humphries, M. R., Conlon, T. J., Spencer, C. T., Poirier, A. E., Garlington, W., Baker, D. J., Song, S., Berns, K. I., et al. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.1177 · Cited by 177 · see citing works

Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models

Hakim, C. H., Kumar, S. R. P., Pérez-López, D. O., Wasala, N. B., Zhang, D., Yue, Y., Teixeira, J., Pan, X., Zhang, K., Million, E. D., et al. (2021) · Nature Communications · doi:10.1038/s41467-021-26830-7 · Cited by 175 · see citing works

Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression

Mueller, C., Chulay, J. D., Trapnell, B. C., Humphries, M., Carey, B., Sandhaus, R. A., McElvaney, N. G., Messina, L., Tang, Q., Rouhani, F. N., et al. (2013) · Journal of Clinical Investigation · doi:10.1172/jci70314 · Cited by 169 · see citing works

Fatal thrombotic microangiopathy case following adeno-associated viral <i>SMN</i> gene therapy

Guillou, J., de Pellegars, A., Porcheret, F., Frémeaux-Bacchi, V., Allain-Launay, E., Debord, C., Denis, M., Péréon, Y., Barnérias, C., Desguerre, I., et al. (2022) · Blood Advances · doi:10.1182/bloodadvances.2021006419 · Cited by 166 · see citing works

Construction and analysis of compact muscle-specific promoters for AAV vectors

Wang, B., Li, J., Fu, F. H., Chen, C., Zhu, X., Zhou, L., Jiang, X. L., & Xiao, X. (2008) · Gene Therapy · doi:10.1038/gt.2008.104 · Cited by 149 · see citing works

B-cell depletion is protective against anti-AAV capsid immune response: a human subject case study

Corti, M., Elder, M., Falk, D., Lawson, L., Smith, B., Nayak, S., Conlon, T., Clément, N., Erger, K., Lavassani, E., et al. (2014) · Molecular Therapy - Methods &amp; Clinical Development · doi:10.1038/mtm.2014.33 · Cited by 140 · see citing works

Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy

Schulz, M., Lévy, D., Petropoulos, C. J., Bashirians, G., Winburn, I. C., Mahn, M., Somanathan, S., Cheng, S. H., & Byrne, B. J. (2023) · Molecular Therapy · doi:10.1016/j.ymthe.2023.01.010 · Cited by 132 · see citing works

Plasmacytoid and conventional dendritic cells cooperate in crosspriming AAV capsid-specific CD8+ T cells

Rogers, G. L., Shirley, J. L., Zolotukhin, I., Kumar, S. R. P., Sherman, A., Perrin, G. Q., Hoffman, B. E., Srivastava, A., Basner-Tschakarjan, E., Wallet, M. A., et al. (2017) · Blood · doi:10.1182/blood-2016-11-751040 · Cited by 131 · see citing works

Adeno-associated virus vectored immunoprophylaxis to prevent HIV in healthy adults: a phase 1 randomised controlled trial

Priddy, F. H., Lewis, D. J. M., Gelderblom, H. C., Hassanin, H., Streatfield, C., LaBranche, C., Hare, J., Cox, J. H., Dally, L., Bendel, D., et al. (2019) · The Lancet HIV · doi:10.1016/s2352-3018(19)30003-7 · Cited by 131 · see citing works

Immune responses to AAV-vectors, the Glybera example from bench to bedside

Ferreira, V., Petry, H., & Salmon, F. (2014) · Frontiers in Immunology · doi:10.3389/fimmu.2014.00082 · Cited by 120 · see citing works

Anti-dystrophin T cell responses in Duchenne muscular dystrophy: prevalence and a glucocorticoid treatment effect

Flanigan, K. M., Campbell, K., Viollet, L., Wang, W., Gomez, A. M., Walker, C. M., & Mendell, J. R. (2013) · Human Gene Therapy · doi:10.1089/hum.2013.092 · Cited by 115 · see citing works

Gene therapy enters the pharma market: The short story of a long journey

Büning, H. (2013) · EMBO Molecular Medicine · doi:10.1002/emmm.201202291 · Cited by 113 · see citing works

Exposure to wild-type AAV drives distinct capsid immunity profiles in humans

Kuranda, K., Jean-Alphonse, P., Leborgne, C., Hardet, R., Collaud, F., Marmier, S., Costa Verdera, H., Ronzitti, G., Veron, P., & Mingozzi, F. (2018) · Journal of Clinical Investigation · doi:10.1172/jci122372 · Cited by 104 · see citing works

Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning

Corti, M., Cleaver, B. D., Clément, N., Conlon, T. J., Faris, K. J., Wang, G., Benson, J. M., Tarantal, A. F., Fuller, D., Herzog, R. W., et al. (2015) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2015.068 · Cited by 98 · see citing works

Systemic protein delivery by muscle-gene transfer is limited by a local immune response

Wang, L., Dobrzynski, E., Schlachterman, A., Cao, O., & Herzog, R. W. (2005) · Blood · doi:10.1182/blood-2004-03-0848 · Cited by 94 · see citing works

A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C

Herson, S., Hentati, F., Rigolet, A., Béhin, A., Romero, N. B., Leturcq, F., Laforêt, P., Maisonobe, T., Amouri, R., Haddad, H., et al. (2011) · Brain · doi:10.1093/brain/awr342 · Cited by 91 · see citing works

Re:”Moving forward after two deaths in a gene therapy trial of myotubular myopathy” by Wilson and flotte

Shieh, P. B., Bönnemann, C. G., Müller-Felber, W., Blaschek, A., Dowling, J. J., Kuntz, N. L., & Seferian, A. M. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.217 · Cited by 91 · see citing works

Pre-existing humoral immunity and complement pathway contribute to immunogenicity of adeno-associated virus (AAV) vector in human blood

Smith, C. J., Ross, N., Kamal, A., Kim, K. Y., Kropf, E., Deschatelets, P., Francois, C., Quinn, W. J., Singh, I., Majowicz, A., et al. (2022) · Frontiers in Immunology · doi:10.3389/fimmu.2022.999021 · Cited by 86 · cited 1× · see citing works

Unique Roles of TLR9- and MyD88-Dependent and -Independent Pathways in Adaptive Immune Responses to AAV-Mediated Gene Transfer

Rogers, G. L., Suzuki, M., Zolotukhin, I., Markusic, D. M., Morel, L. M., Lee, B., Ertl, H. C. J., & Herzog, R. W. (2015) · Journal of Innate Immunity · doi:10.1159/000369273 · Cited by 85 · see citing works

Effect of CpG Depletion of Vector Genome on CD8+ T Cell Responses in AAV Gene Therapy

Bertolini, T. B., Shirley, J. L., Zolotukhin, I., Li, X., Kaisho, T., Xiao, W., Kumar, S. R. P., & Herzog, R. W. (2021) · Frontiers in Immunology · doi:10.3389/fimmu.2021.672449 · Cited by 80 · see citing works

Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency

Calcedo, R., Somanathan, S., Qin, Q., Betts, M. R., Rech, A. J., Vonderheide, R. H., Mueller, C., Flotte, T. R., & Wilson, J. M. (2017) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1617726114 · Cited by 79 · see citing works

Type I IFN Sensing by cDCs and CD4+ T Cell Help Are Both Requisite for Cross-Priming of AAV Capsid-Specific CD8+ T Cells

Shirley, J. L., Keeler, G. D., Sherman, A., Zolotukhin, I., Markusic, D. M., Hoffman, B. E., Morel, L. M., Wallet, M. A., Terhorst, C., & Herzog, R. W. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2019.11.011 · Cited by 79 · see citing works

Distinct immune responses to transgene products from rAAV1 and rAAV8 vectors

Lu, Y., & Song, S. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0909520106 · Cited by 65 · see citing works

Prolonged Gene Expression in Muscle Is Achieved Without Active Immune Tolerance Using MicrorRNA 142.3p-Regulated rAAV Gene Transfer

Boisgerault, F., Gross, D.-A., Ferrand, M., Poupiot, J., Darocha, S., Richard, I., & Galy, A. (2013) · Human Gene Therapy · doi:10.1089/hum.2012.208 · Cited by 63 · see citing works

Complexity of immune responses to AAV transgene products – Example of factor IX

Herzog, R. W. (2019) · Cellular Immunology · doi:10.1016/j.cellimm.2017.05.006 · Cited by 60 · see citing works

Fourth Boy Dies in Clinical Trial of Astellas' AT132

Philippidis, A. (2021) · Human Gene Therapy · doi:10.1089/hum.2021.29182.bfs · Cited by 59 · see citing works

Meeting Report: 2022 Muscular Dystrophy Association Summit on ‘Safety and Challenges in Gene Transfer Therapy’

Lek, A., Atas, E., Hesterlee, S. E., Byrne, B. J., & Bönnemann, C. G. (2023) · Journal of Neuromuscular Diseases · doi:10.3233/jnd-221639 · Cited by 59 · see citing works

Major Subsets of Human Dendritic Cells Are Efficiently Transduced by Self-Complementary Adeno-Associated Virus Vectors 1 and 2

Véron, P., Allo, V., Rivière, C., Bernard, J., Douar, A., & Masurier, C. (2007) · Journal of Virology · doi:10.1128/jvi.02516-06 · Cited by 54 · see citing works

Therapeutic Levels of Factor IX Expression Using a Muscle-Specific Promoter and Adeno-Associated Virus Serotype 1 Vector

Liu, Y.-L., Mingozzi, F., Rodriguez-Colon, S. M., Joseph, S. O., Dobrzynski, E., Suzuki, T., High, K. A., & Herzog, R. W. (2004) · Human Gene Therapy · doi:10.1089/1043034041648453 · Cited by 54 · see citing works

Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity

Xiao, Y., Muhuri, M., Li, S., Qin, W., Xu, G., Luo, L., Li, J., Letizia, A. J., Wang, S. K., Chan, Y. K., et al. (2019) · JCI Insight · doi:10.1172/jci.insight.99052 · Cited by 53 · see citing works

Rational engineering of a functional CpG-free ITR for AAV gene therapy

Pan, X., Yue, Y., Boftsi, M., Wasala, L. P., Tran, N. T., Zhang, K., Pintel, D. J., Tai, P. W. L., & Duan, D. (2022) · Gene Therapy · doi:10.1038/s41434-021-00296-0 · Cited by 53 · see citing works

Intramuscular administration of AAV overcomes pre-existing neutralizing antibodies in rhesus macaques

Greig, J. A., Calcedo, R., Grant, R. L., Peng, H., Medina-Jaszek, C. A., Ahonkhai, O., Qin, Q., Roy, S., Tretiakova, A. P., & Wilson, J. M. (2016) · Vaccine · doi:10.1016/j.vaccine.2016.10.053 · Cited by 52 · see citing works

Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction

Muhuri, M., Zhan, W., Maeda, Y., Li, J., Lotun, A., Chen, J., Sylvia, K., Dasgupta, I., Arjomandnejad, M., Nixon, T., et al. (2021) · Frontiers in Immunology · doi:10.3389/fimmu.2021.674242 · Cited by 50 · see citing works

Rapid development of genotypic resistance to lamivudine when combined with zidovudine in pregnancy

Clarke, J. R., Braganza, R., Mirza, A., Stainsby, C. M., Ait‐Khaled, M., Wright, A., Lyall, H., Parker, D., McClure, M. O., Weber, J. N., et al. (1999) · Journal of Medical Virology · doi:10.1002/(sici)1096-9071(199911)59:3 · Cited by 44 · see citing works

TLR9-Activating CpG-B ODN but Not TLR7 Agonists Triggers Antibody Formation to Factor IX in Muscle Gene Transfer

Butterfield, J. S. S., Biswas, M., Shirley, J. L., Kumar, S. R. P., Sherman, A., Terhorst, C., Ling, C., & Herzog, R. W. (2019) · Human Gene Therapy Methods · doi:10.1089/hgtb.2019.013 · Cited by 35 · see citing works

Hemophagocytic lymphohistiocytosis following gene replacement therapy in a child with type 1 spinal muscular atrophy

Galletta, F., Cucinotta, U., Marseglia, L. M., Cacciola, A., Gallizzi, R., Cuzzocrea, S., Messina, S., Toscano, A., & Gitto, E. (2022) · Journal of Clinical Pharmacy and Therapeutics · doi:10.1111/jcpt.13733 · Cited by 35 · see citing works

Structural and functional characterization of capsid binding by anti-AAV9 monoclonal antibodies from infants after SMA gene therapy

Logan, G. J., Mietzsch, M., Khandekar, N., D’Silva, A., Anderson, D., Mandwie, M., Hsi, J., Nelson, A. R., Chipman, P. R., Jackson, J., et al. (2023) · Molecular Therapy · doi:10.1016/j.ymthe.2023.03.032 · Cited by 28 · see citing works

Immune transgene-dependent myocarditis in macaques after systemic administration of adeno-associated virus expressing human acid alpha-glucosidase

Hordeaux, J., Ramezani, A., Tuske, S., Mehta, N., Song, C., Lynch, A., Lupino, K., Chichester, J. A., Buza, E. L., Dyer, C., et al. (2023) · Frontiers in Immunology · doi:10.3389/fimmu.2023.1094279 · Cited by 23 · see citing works

Neonatal Fc Receptor Inhibition Enables Adeno-Associated Virus Gene Therapy Despite Pre-Existing Humoral Immunity

Horiuchi, M., Hinderer, C. J., Shankle, H. N., Hayashi, P. M., Chichester, J. A., Kissel, C. L., Bell, P. L., Dyer, C., & Wilson, J. M. (2023) · Human Gene Therapy · doi:10.1089/hum.2022.216 · Cited by 21 · see citing works

Two gene therapies for hemophilia available: Now what?

Pierce, G. F., & Herzog, R. W. (2023) · Molecular Therapy · doi:10.1016/j.ymthe.2023.03.001 · Cited by 21 · see citing works

Secondary hemophagocytic lymphohistiocytosis following Zolgensma therapy: An evolving story on the innate response to systemic gene therapy.

Byrne, B., Elder, M., Leon-Astudillo, C., & Corti, M. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.11.007 · Cited by 15 · see citing works

T Cell Responses to Dystrophin in a Natural History Study of Duchenne Muscular Dystrophy

Anthony, K., Ala, P., Catapano, F., et al. (2022) · Human Gene Therapy · doi:10.1089/hum.2022.166 · Cited by 12 · see citing works

Neutralizing the Neutralizers in AAV Gene Therapy

Herzog, R. W., & Biswas, M. (2020) · Molecular Therapy · doi:10.1016/j.ymthe.2020.07.015 · Cited by 10 · see citing works

Assessment of the Gene Therapy Immune Response in the Canine Muscular Dystrophy Model

Hakim, C. H., Kumar, S. R. P., Pérez‐López, D. O., et al. (2022) · Methods in molecular biology · doi:10.1007/978-1-0716-2772-3_18 · Cited by 7 · see citing works

Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne’s Muscular Dystrophy

Lek, A., Wong, B., Keeler, A., Blackwood, M., Ma, K., Huang, S., Sylvia, K., Batista, A. R., Artinian, R., Kokoski, D., et al. (2023) · New England Journal of Medicine · doi:10.1056/nejmoa2307798 · Cited by 319 · see citing works

Lethal immunotoxicity in high-dose systemic AAV therapy

Duan, D. (2023) · Molecular Therapy · doi:10.1016/j.ymthe.2023.10.015 · Cited by 240 · see citing works

High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury

Hordeaux, J., Lamontagne, R. J., Song, C., Buchlis, G., Dyer, C., Buza, E. L., Ramezani, A., Wielechowski, E., Greig, J. A., Chichester, J. A., et al. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.02.002 · Cited by 81 · see citing works

Innate immune sensing of adeno-associated virus vectors

Cao, D., Byrne, B. J., de Jong, Y. P., Terhorst, C., Duan, D., Herzog, R. W., & Kumar, S. R. P. (2024) · Human Gene Therapy · doi:10.1089/hum.2024.040 · Cited by 50 · see citing works

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

Lam, A. K., Frabutt, D. A., Li, L., & Xiao, W. (2021) · Human Gene Therapy · doi:10.1089/hum.2021.124 · Cited by 25 · see citing works

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

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

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