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

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

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

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

Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector

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Antigen-presenting cell function in the tolerogenic liver environment

Thomson, A. W., & Knolle, P. A. (2010) · Nature reviews. Immunology · doi:10.1038/nri2858 · Cited by 804 · 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

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

Recombinant adeno-associated virus for muscle directed gene therapy

Fisher, K. J., Jooss, K., Alston, J., Yang, Y., Haecker, S. E., High, K., Pathak, R., Raper, S. E., & Wilson, J. M. (1997) · Nature Medicine · doi:10.1038/nm0397-306 · Cited by 654 · see citing works

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

Kessler, P., Podsakoff, G. M., Chen, X., McQuiston, S. A., Colosi, P., Matelis, L. A., Kurtzman, G. J., & Byrne, B. J. (1996) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.93.24.14082 · Cited by 614 · see citing works

Transduction of Dendritic Cells by DNA Viral Vectors Directs the Immune Response to Transgene Products in Muscle Fibers

Jooss, K., Yang, Y., Fisher, K. J., & Wilson, J. M. (1998) · Journal of Virology · doi:10.1128/jvi.72.5.4212-4223.1998 · Cited by 470 · see citing works

Induction of Immune Tolerance to Coagulation Factor IX Antigen by In Vivo Hepatic Gene Transfer

Mingozzi, F., Liu, Y.-L., Dobrzynski, E., Kaufhold, A., Liu, J. H., Wang, Y., Arruda, V. R., High, K. A., & Herzog, R. W. (2003) · Journal of Clinical Investigation · doi:10.1172/jci16887 · Cited by 466 · see citing works

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

Bennett, J., Wellman, J., Marshall, K. A., McCague, S., Ashtari, M., DiStefano-Pappas, J., Elci, O. U., Chung, D. C., Sun, J., Wright, J. F., et al. (2016) · The Lancet · doi:10.1016/s0140-6736(16)30371-3 · Cited by 461 · see citing works

The Indoleamine 2,3-Dioxygenase Pathway Is Essential for Human Plasmacytoid Dendritic Cell-Induced Adaptive T Regulatory Cell Generation

Chen, W., Liang, X., Peterson, A. J., Munn, D. H., & Blazar, B. R. (2008) · The Journal of Immunology · doi:10.4049/jimmunol.181.8.5396 · Cited by 453 · see citing works

Polymeric synthetic nanoparticles for the induction of antigen-specific immunological tolerance

Maldonado, R. A., LaMothe, R. A., Ferrari, J. D., Zhang, A.-H., Rossi, R. J., Kolte, P. N., Griset, A. P., O’Neil, C., Altreuter, D. H., Browning, E., et al. (2015) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1408686111 · Cited by 441 · 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

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

Bennett, J., Ashtari, M., Wellman, J., Marshall, K. A., Cyckowski, L. L., Chung, D. C., McCague, S., Pierce, E. A., Chen, Y., Bennicelli, J. L., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3002865 · Cited by 395 · see citing works

In vivo tissue-tropism of adeno-associated viral vectors

Srivastava, A. (2016) · Current Opinion in Virology · doi:10.1016/j.coviro.2016.08.003 · Cited by 346 · see citing works

Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

Nathwani, A. C., Rosales, C., McIntosh, J., Rastegarlari, G., Nathwani, D., Raj, D., Nawathe, S., Waddington, S. N., Bronson, R., Jackson, S., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2010.274 · Cited by 345 · see citing works

Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy

Niemeyer, G. P., Herzog, R. W., Mount, J., Arruda, V. R., Tillson, D. M., Hathcock, J., van Ginkel, F. W., High, K. A., & Lothrop, C. D. (2009) · Blood · doi:10.1182/blood-2008-10-181479 · Cited by 276 · see citing works

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

Mingozzi, F., Hasbrouck, N. C., Basner-Tschakarjan, E., Edmonson, S. A., Hui, D. J., Sabatino, D. E., Zhou, S., Wright, J. F., Jiang, H., Pierce, G. F., et al. (2007) · Blood · doi:10.1182/blood-2007-03-080093 · Cited by 254 · 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

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

Hepatic regulatory T cells and Kupffer cells are crucial mediators of systemic T cell tolerance to antigens targeting murine liver

Breous, E., Somanathan, S., Vandenberghe, L. H., & Wilson, J. M. (2009) · Hepatology · doi:10.1002/hep.23043 · Cited by 237 · see citing works

Clinical development of gene therapy: results and lessons from recent successes

Kumar, S. R. P., Markusic, D. M., Biswas, M., High, K. A., & Herzog, R. W. (2016) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2016.34 · Cited by 235 · see citing works

Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer

Cao, O., Dobrzynski, E., Wang, L., Nayak, S., Mingle, B., Terhorst, C., & Herzog, R. W. (2007) · Blood · doi:10.1182/blood-2007-02-073304 · Cited by 220 · see citing works

Inhibitors of Factor VIII in Black Patients with Hemophilia

Viel, K. R., Ameri, A., Abshire, T. C., Iyer, R. V., Watts, R. G., Lutcher, C. L., Channell, C., Cole, S. A., Fernstrom, K. M., Nakaya, S. M., et al. (2009) · New England Journal of Medicine · doi:10.1056/nejmoa075760 · Cited by 199 · see citing works

Muscle-Directed Gene Transfer and Transient Immune Suppression Result in Sustained Partial Correction of Canine Hemophilia B Caused by a Null Mutation

Herzog, R. W., Mount, J. D., Arruda, V. R., High, K. A., & Lothrop, C. D. (2001) · Molecular Therapy · doi:10.1006/mthe.2001.0442 · Cited by 176 · 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

Engineered antigen-specific human regulatory T cells: immunosuppression of FVIII-specific T- and B-cell responses

Kim, Y. C., Zhang, A.-H., Su, Y. A., Rieder, S. A., Rossi, R. J., Ettinger, R. A., Pratt, K. P., Shevach, E. M., & Scott, D. W. (2014) · Blood · doi:10.1182/blood-2014-04-566786 · Cited by 160 · see citing works

Eradication of neutralizing antibodies to factor VIII in canine hemophilia a after liver gene therapy

Finn, J. D., Ozelo, M. C., Sabatino, D. E., Franck, H. W. G., Merricks, E. P., Crudele, J. M., Zhou, S., Kazazian, H. H., Lillicrap, D., Nichols, T. C., et al. (2010) · Blood · doi:10.1182/blood-2010-06-288001 · Cited by 157 · see citing works

AAV liver expression of FIX-padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia b dogs and mice

Crudele, J. M., Finn, J. D., Siner, J. I., Martin, N. B., Niemeyer, G. P., Zhou, S., Mingozzi, F., Lothrop, C. D., & Arruda, V. R. (2015) · Blood · doi:10.1182/blood-2014-07-588194 · Cited by 153 · see citing works

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

Arruda, V. R., Stedman, H. H., Nichols, T. C., Haskins, M. E., Nicholson, M., Herzog, R. W., Couto, L. B., & High, K. A. (2004) · Blood · doi:10.1182/blood-2004-07-2908 · Cited by 153 · see citing works

FVIII-specific human chimeric antigen receptor T-regulatory cells suppress T- and B-cell responses to FVIII

Yoon, J., Schmidt, A., Zhang, A.-H., Königs, C., Kim, Y. C., & Scott, D. W. (2017) · Blood · doi:10.1182/blood-2016-07-727834 · Cited by 150 · see citing works

Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1

Arruda, V. R., Schüttrumpf, J., Herzog, R. W., Nichols, T. C., Robinson, N. A., Lotfi, Y., Mingozzi, F., Xiao, W., Couto, L. B., & High, K. A. (2003) · Blood · doi:10.1182/blood-2003-05-1446 · Cited by 149 · see citing works

Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy

Herzog, R. W., Fields, P. A., Arruda, V. R., Brubaker, J. O., Armstrong, E., McClintock, D., Bellinger, D. A., Couto, L. B., Nichols, T. C., & High, K. A. (2002) · Human Gene Therapy · doi:10.1089/104303402760128513 · Cited by 147 · 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 & Clinical Development · doi:10.1038/mtm.2014.33 · Cited by 140 · see citing works

Induction of antigen-specific CD4+ T-cell anergy and deletion by in vivo viral gene transfer

Dobrzynski, E., Mingozzi, F., Liu, Y.-L., Bendo, E., Cao, O., Wang, L., & Herzog, R. W. (2004) · Blood · doi:10.1182/blood-2004-03-0847 · Cited by 135 · see citing works

Role of Vector in Activation of T Cell Subsets in Immune Responses against the Secreted Transgene Product Factor IX

Fields, P. A., Kowalczyk, D. W., Arruda, V. R., Armstrong, E., McCleland, M. L., Hagstrom, J. N., Pasi, J., Ertl, H. C. J., Herzog, R. W., & High, K. A. (2000) · Molecular Therapy · doi:10.1006/mthe.2000.0032 · Cited by 129 · see citing works

Risk and Prevention of Anti-factor IX Formation in AAV-Mediated Gene Transfer in the Context of a Large Deletion of F9

Fields, P. A., Arruda, V. R., Armstrong, E., Chu, K., Mingozzi, F., Hagstrom, J. N., Herzog, R. W., & High, K. A. (2001) · Molecular Therapy · doi:10.1006/mthe.2001.0441 · Cited by 127 · see citing works

The Complex and Evolving Story of T cell Activation to AAV Vector-encoded Transgene Products

Mays, L. E., & Wilson, J. M. (2010) · Molecular Therapy · doi:10.1038/mt.2010.250 · Cited by 126 · see citing works

The miR-126-VEGFR2 axis controls the innate response to pathogen-associated nucleic acids

Agudo, J., Ruzo, A., Tung, N., Salmon, H., Leboeuf, M., Hashimoto, D., Becker, C., Garrett-Sinha, L.-A., Baccarini, A., Merad, M., et al. (2014) · Nature Immunology · doi:10.1038/ni.2767 · Cited by 122 · see citing works

Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

Markusic, D. M., Hoffman, B. E., Perrin, G. Q., Nayak, S., Wang, X., LoDuca, P. A., High, K. A., & Herzog, R. W. (2013) · EMBO Molecular Medicine · doi:10.1002/emmm.201302859 · Cited by 116 · see citing works

Expansion of peripheral naturally occurring T regulatory cells by Fms-like tyrosine kinase 3 ligand treatment

Swee, L. K., Bosco, N., MALISSEN, B., Ceredig, R. H., & Rolink, A. G. (2009) · Blood · doi:10.1182/blood-2008-06-161026 · Cited by 116 · see citing works

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B

Arruda, V. R., Stedman, H. H., Haurigot, V., Buchlis, G., Baila, S., Favaro, P. M. B., Chen, Y.-F., Franck, H. W. G., Zhou, S., Wright, J. F., et al. (2010) · Blood · doi:10.1182/blood-2009-12-261156 · Cited by 115 · 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

Hepatic Gene Transfer as a Means of Tolerance Induction to Transgene Products

LoDuca, P. A., Hoffman, B. E., & Herzog, R. W. (2009) · Current Gene Therapy · doi:10.2174/156652309787909490 · Cited by 114 · see citing works

Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid

Vercauteren, K., Hoffman, B. E., Zolotukhin, I., Keeler, G. D., Xiao, J. W., Basner-Tschakarjan, E., High, K. A., Ertl, H. C., Rice, C. M., Srivastava, A., et al. (2016) · Molecular Therapy · doi:10.1038/mt.2016.61 · Cited by 114 · see citing works

Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter

Sun, B., Zhang, H., Franco, L. M., Brown, T. T., Bird, A., Schneider, A., & Koeberl, D. D. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.01.012 · Cited by 111 · see citing works

The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy

Finn, J. D., Nichols, T. C., Svoronos, N., Merricks, E. P., Bellenger, D. A., Zhou, S., Simioni, P., High, K. A., & Arruda, V. R. (2012) · Blood · doi:10.1182/blood-2012-06-440123 · Cited by 109 · see citing works

Prevention of cytotoxic T lymphocyte responses to factor IX-expressing hepatocytes by gene transfer-induced regulatory T cells

Dobrzynski, E., Fitzgerald, J. C., Cao, O., Mingozzi, F., Wang, L., & Herzog, R. W. (2006) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0508685103 · Cited by 105 · see citing works

Gene Therapy for Hemophilia

Nienhuis, A. W., Nathwani, A. C., & Davidoff, A. M. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.03.033 · Cited by 99 · 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

Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions

Basner‐Tschakarjan, E., & Mingozzi, F. (2014) · Frontiers in Immunology · doi:10.3389/fimmu.2014.00350 · Cited by 95 · see citing works

Major role of local immune responses in antibody formation to factor IX in AAV gene transfer

Wang, L., Cao, O., Swalm, B., Dobrzynski, E., Mingozzi, F., & Herzog, R. W. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302539 · Cited by 95 · 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

Impact of the Underlying Mutation and the Route of Vector Administration on Immune Responses to Factor IX in Gene Therapy for Hemophilia B

Cao, O., Hoffman, B. E., Moghimi, B., Nayak, S., Cooper, M., Zhou, S., Ertl, H. C., High, K. A., & Herzog, R. W. (2009) · Molecular Therapy · doi:10.1038/mt.2009.159 · Cited by 92 · see citing works

Mechanism of oral tolerance induction to therapeutic proteins

Wang, X., Sherman, A., Liao, G., Leong, K. W., Daniell, H., Terhorst, C., & Herzog, R. W. (2012) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2012.10.013 · Cited by 89 · see citing works

Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease

Zhang, P., Sun, B., Osada, T., Rodriguiz, R., Yang, X. Y., Luo, X., Kemper, A. R., Clay, T. M., & Koeberl, D. D. (2012) · Human Gene Therapy · doi:10.1089/hum.2011.063 · Cited by 88 · see citing works

Improved Induction of Immune Tolerance to Factor IX by Hepatic AAV-8 Gene Transfer

Cooper, M., Nayak, S., Hoffman, B. E., Terhorst, C., Cao, O., & Herzog, R. W. (2009) · Human Gene Therapy · doi:10.1089/hum.2008.161 · Cited by 88 · see citing works

Recombinant Adeno-Associated Virus Vectors Induce Functionally Impaired Transgene Product-Specific CD8+ T Cells in Mice

Lin, S.-W., Hensley, S. E., Tatsis, N., Lasaro, M. O., & Ertl, H. C. J. (2007) · Journal of Clinical Investigation · doi:10.1172/jci33138 · Cited by 88 · 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

The Balance between CD8(+) T Cell-Mediated Clearance of AAV-Encoded Antigen in the Liver and Tolerance Is Dependent on the Vector Dose

Kumar, S. R. P., Hoffman, B. E., Terhorst, C., de Jong, Y. P., & Herzog, R. W. (2017) · Molecular Therapy · doi:10.1016/j.ymthe.2017.02.014 · Cited by 83 · see citing works

B-Cell Depletion and Immunomodulation before Initiation of Enzyme Replacement Therapy Blocks the Immune Response to Acid Alpha-Glucosidase in Infantile-Onset Pompe Disease

Elder, M. E., Nayak, S., Collins, S. W., Lawson, L. A., Kelley, J. S., Herzog, R. W., Modica, R., Lew, J., Lawrence, R. M., & Byrne, B. J. (2013) · The Journal of Pediatrics · doi:10.1016/j.jpeds.2013.03.002 · Cited by 81 · 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

Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice

Lange, A. M., Altynova, E. S., Nguyen, G. N., & Sabatino, D. E. (2016) · Molecular Therapy - Methods & Clinical Development · doi:10.1038/mtm.2016.64 · Cited by 78 · see citing works

Low-dose liver-targeted gene therapy for pompe disease enhances therapeutic efficacy of ERT via immune tolerance induction

Han, S.-O., Ronzitti, G., Arnson, B., Leborgne, C., Li, S., Mingozzi, F., & Koeberl, D. (2017) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2016.12.010 · Cited by 78 · see citing works

Transient B Cell Depletion or Improved Transgene Expression by Codon Optimization Promote Tolerance to Factor VIII in Gene Therapy

Sack, B. K., Merchant, S. N., Markusic, D. M., Nathwani, A. C., Davidoff, A. M., Byrne, B. J., & Herzog, R. W. (2012) · PLoS ONE · doi:10.1371/journal.pone.0037671 · Cited by 75 · see citing works

Nonredundant Roles of IL-10 and TGF-β in Suppression of Immune Responses to Hepatic AAV-Factor IX Gene Transfer

Hoffman, B. E., Martino, A. T., Sack, B. K., Cao, O., Liao, G., Terhorst, C., & Herzog, R. W. (2011) · Molecular Therapy · doi:10.1038/mt.2011.33 · Cited by 72 · see citing works

Development of gene transfer for induction of antigen-specific tolerance

Sack, B. K., Herzog, R. W., Terhorst, C., & Markusic, D. M. (2014) · Molecular Therapy - Methods & Clinical Development · doi:10.1038/mtm.2014.13 · Cited by 70 · see citing works

Synergy between rapamycin and FLT3 ligand enhances plasmacytoid dendritic cell-dependent induction of CD4+CD25+FoxP3+ Treg

Biswas, M., Sarkar, D., Kumar, S. R. P., Nayak, S., Rogers, G. L., Markusic, D. M., Liao, G., Terhorst, C., & Herzog, R. W. (2015) · Blood · doi:10.1182/blood-2014-09-599266 · Cited by 70 · see citing works

Mir‐142‐3p target sequences reduce transgene‐directed immunogenicity following intramuscular adeno‐associated virus 1 vector‐mediated gene delivery

Majowicz, A., Maczuga, P., Kwikkers, K. L., van der Marel, S., van Logtenstein, R., Petry, H., van Deventer, S. J. H., Konstantinova, P. S., & Ferreira, V. (2013) · The Journal of Gene Medicine · doi:10.1002/jgm.2712 · Cited by 69 · see citing works

Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse

Passini, M. A., Bu, J., Fidler, J. A., Ziegler, R. J., Foley, J. W., Dodge, J. C., Yang, W. W., Clarke, J. L., Taksir, T. V., Griffiths, D. A., et al. (2007) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0703509104 · Cited by 67 · see citing works

Insulin B chain 9–23 gene transfer to hepatocytes protects from type 1 diabetes by inducing Ag-specific FoxP3 + T regs

Akbarpour, M., Goudy, K., Cantore, A., Russo, F., Sanvito, F., Naldini, L., Annoni, A., & Roncarolo, M. G. (2015) · Science Translational Medicine · doi:10.1126/scitranslmed.aaa3032 · Cited by 66 · see citing works

Prophylactic immune tolerance induced by changing the ratio of antigen-specific effector to regulatory T cells

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Self-complementary AAVs Induce More Potent Transgene Product-specific Immune Responses Compared to a Single-stranded Genome

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Plant cell-made protein antigens for induction of Oral tolerance

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Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins

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Additional file 1 of Truncated mini LRP1 transports cargo from luminal to basolateral side across the blood brain barrier

Fritzen, L., Wienken, K., Wagner, L., Kurtyka, M., Vogel, K., Körbelin, J., Weggen, S., Fricker, G., & Pietrzik, C. U. (2024) · Figshare · doi:10.6084/m9.figshare.27049039

Additional file 6 of Truncated mini LRP1 transports cargo from luminal to basolateral side across the blood brain barrier

Fritzen, L., Wienken, K., Wagner, L., Kurtyka, M., Vogel, K., Körbelin, J., Weggen, S., Fricker, G., & Pietrzik, C. U. (2024) · Figshare · doi:10.6084/m9.figshare.27049054

Additional file 8 of Truncated mini LRP1 transports cargo from luminal to basolateral side across the blood brain barrier

Fritzen, L., Wienken, K., Wagner, L., Kurtyka, M., Vogel, K., Körbelin, J., Weggen, S., Fricker, G., & Pietrzik, C. U. (2024) · Figshare · doi:10.6084/m9.figshare.27049060

Additional file 4 of Truncated mini LRP1 transports cargo from luminal to basolateral side across the blood brain barrier

Fritzen, L., Wienken, K., Wagner, L., Kurtyka, M., Vogel, K., Körbelin, J., Weggen, S., Fricker, G., & Pietrzik, C. U. (2024) · Figshare · doi:10.6084/m9.figshare.27049048

Additional file 5 of Truncated mini LRP1 transports cargo from luminal to basolateral side across the blood brain barrier

Fritzen, L., Wienken, K., Wagner, L., Kurtyka, M., Vogel, K., Körbelin, J., Weggen, S., Fricker, G., & Pietrzik, C. U. (2024) · Figshare · doi:10.6084/m9.figshare.27049051

Additional file 3 of Truncated mini LRP1 transports cargo from luminal to basolateral side across the blood brain barrier

Fritzen, L., Wienken, K., Wagner, L., Kurtyka, M., Vogel, K., Körbelin, J., Weggen, S., Fricker, G., & Pietrzik, C. U. (2024) · Figshare · doi:10.6084/m9.figshare.27049045

Additional file 9 of Truncated mini LRP1 transports cargo from luminal to basolateral side across the blood brain barrier

Fritzen, L., Wienken, K., Wagner, L., Kurtyka, M., Vogel, K., Körbelin, J., Weggen, S., Fricker, G., & Pietrzik, C. U. (2024) · Figshare · doi:10.6084/m9.figshare.27049063

Additional file 2 of Truncated mini LRP1 transports cargo from luminal to basolateral side across the blood brain barrier

Fritzen, L., Wienken, K., Wagner, L., Kurtyka, M., Vogel, K., Körbelin, J., Weggen, S., Fricker, G., & Pietrzik, C. U. (2024) · Figshare · doi:10.6084/m9.figshare.27049042

Additional file 7 of Truncated mini LRP1 transports cargo from luminal to basolateral side across the blood brain barrier

Fritzen, L., Wienken, K., Wagner, L., Kurtyka, M., Vogel, K., Körbelin, J., Weggen, S., Fricker, G., & Pietrzik, C. U. (2024) · Figshare · doi:10.6084/m9.figshare.27049057

Adeno-associated virus-induced neurotoxicity is prevented by CpG depletion

Dunn, C. E., Carvajal, B. V., Ma, J., Castaneda-Hernandez, G., Nguyen, A. V., Jung, C., Suriano, C. M., & Boulanger, L. M. (2026) · Molecular Therapy Advances · doi:10.1016/j.omta.2026.201703

Balance entre rejet et tolérance : la bipolarité immunitaire du muscle squelettique face à l’AAV recombinant : décryptage de la réponse immune de l’hôte dirigée contre le produit du transgène après injection de vecteurs AAV recombinants par voie intramusculaire

Journou, M. (2021) · theses.fr (ABES) · doi:10.70675/9a618ac2za0a4z4e90zbdb3ze14482ffa655

Hepatic Tolerance Induction Prevents Inflammatory Responses To Factor IX Expressing Cells at Supplementary Sites of Gene Transfer.

Dobrzynski, E., Mingozzi, F., Wang, L.-S., Mingle, B., Cao, O., & Herzog, R. W. (2004) · Blood · doi:10.1182/blood.v104.11.3185.3185

Hepatic AAV Gene Transfer and the Immune System: Friends or Foes?

Herzog, R. W. (2010) · Molecular Therapy · doi:10.1038/mt.2010.96 · Cited by 14 · see citing works

Major role of local immune responses in antibody formation to factor IX in AAV gene transfer

Wang, L., Cao, O., Swalm, B., Dobrzynski, E., Mingozzi, F., & Herzog, R. W. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302539 · Cited by 95 · see citing works

Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette

Kumar, S. R. P., Xie, J., Hu, S., Ko, J., Huang, Q., Brown, H. C., Srivastava, A., Markusic, D. M., Doering, C. B., Spencer, H. T., et al. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.08.001 · Cited by 16 · see citing works

AAV-Mediated Gene Transfer for Treatment of Hemophilia

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