Publications

  1. Yin L, Keeler GD, Zhang Y, Hoffman BE, Ling C, Qing K, Srivastava A. AAV3-miRNA vectors for growth suppression of human hepatocellular carcinoma cells in vitro and human liver tumors in a murine xenograft model in vivo. Gene Ther. 2021 Aug;28(7-8):422-434. doi: 10.1038/s41434-020-0140-1. Epub 2020 Mar 9. PubMed PMID: 32152434; PubMed Central PMCID: PMC7784898.
  2. Yang H, Qing K, Keeler GD, Yin L, Mietzsch M, Ling C, Hoffman BE, Agbandje-McKenna M, Tan M, Wang W, Srivastava A. Enhanced Transduction of Human Hematopoietic Stem Cells by AAV6 Vectors: Implications in Gene Therapy and Genome Editing. Mol Ther Nucleic Acids. 2020 Jun 5;20:451-458. doi: 10.1016/j.omtn.2020.03.009. Epub 2020 Mar 29. PubMed PMID: 32276210; PubMed Central PMCID: PMC7150427.
  3. Shirley JL, Keeler GD, Sherman A, Zolotukhin I, Markusic DM, Hoffman BE, Morel LM, Wallet MA, Terhorst C, Herzog RW. Type I IFN Sensing by cDCs and CD4+ T Cell Help Are Both Requisite for Cross-Priming of AAV Capsid-Specific CD8+ T Cells. Mol Ther. 2020 Mar 4;28(3):758-770. doi: 10.1016/j.ymthe.2019.11.011. Epub 2019 Nov 15. PubMed PMID: 31780366; PubMed Central PMCID: PMC7054715.
  4. Keeler GD, Markusic DM, Hoffman BE. Liver induced transgene tolerance with AAV vectors. Cell Immunol. 2019 Aug;342:103728. doi: 10.1016/j.cellimm.2017.12.002. Epub 2017 Dec 5. Review. PubMed PMID: 29576315; PubMed Central PMCID: PMC5988960.
  5. Herzog RW, Cooper M, Perrin GQ, Biswas M, Martino AT, Morel L, Terhorst C, Hoffman BE. Regulatory T cells and TLR9 activation shape antibody formation to a secreted transgene product in AAV muscle gene transfer. Cell Immunol. 2019 Aug;342:103682. doi: 10.1016/j.cellimm.2017.07.012. Epub 2017 Aug 1. PubMed PMID: 28888664; PubMed Central PMCID: PMC5794662.
  6. Keeler GD, Kumar S, Palaschak B, Silverberg EL, Markusic DM, Jones NT, Hoffman BE. Gene Therapy-Induced Antigen-Specific Tregs Inhibit Neuro-inflammation and Reverse Disease in a Mouse Model of Multiple Sclerosis. Mol Ther. 2018 Jan 3;26(1):173-183. doi: 10.1016/j.ymthe.2017.09.001. Epub 2017 Sep 21. PubMed PMID: 28943274; PubMed Central PMCID: PMC5762980.
  7. Krotova K, Marek GW, Wang RL, Aslanidi G, Hoffman BE, Khodayari N, Rouhani FN, Brantly ML. Alpha-1 Antitrypsin-Deficient Macrophages Have Increased Matriptase-Mediated Proteolytic Activity. Am J Respir Cell Mol Biol. 2017 Aug;57(2):238-247. doi: 10.1165/rcmb.2016-0366OC. PubMed PMID: 28362108; PubMed Central PMCID: PMC5800887.
  8. Rogers GL, Shirley JL, Zolotukhin I, Kumar SRP, Sherman A, Perrin GQ, Hoffman BE, Srivastava A, Basner-Tschakarjan E, Wallet MA, Terhorst C, Biswas M, Herzog RW. Plasmacytoid and conventional dendritic cells cooperate in crosspriming AAV capsid-specific CD8+ T cells. Blood. 2017 Jun 15;129(24):3184-3195. doi: 10.1182/blood-2016-11-751040. Epub 2017 May 3. PubMed PMID: 28468798; PubMed Central PMCID: PMC5472899.
  9. Kumar SRP, Hoffman BE, Terhorst C, de Jong YP, Herzog RW. The Balance between CD8+ T Cell-Mediated Clearance of AAV-Encoded Antigen in the Liver and Tolerance Is Dependent on the Vector Dose. Mol Ther. 2017 Apr 5;25(4):880-891. doi: 10.1016/j.ymthe.2017.02.014. Epub 2017 Mar 9. PubMed PMID: 28284982; PubMed Central PMCID: PMC5383644.
  10. Geoffrey D Keeler, Brad E Hoffman. Movie S1. A Representative Video Documenting the Disease Sequela and Reversal of Clinical Symptoms following AAV Immunotherapy. In: 2017. DOI: 10.13140/rg.2.2.25895.44964.
  11. Zolotukhin I, Markusic DM, Palaschak B, Hoffman BE, Srikanthan MA, Herzog RW. Potential for cellular stress response to hepatic factor VIII expression from AAV vector. Mol Ther Methods Clin Dev. 2016;3:16063. doi: 10.1038/mtm.2016.63. eCollection 2016. PubMed PMID: 27738644; PubMed Central PMCID: PMC5040172.
  12. Vercauteren K, Hoffman BE, Zolotukhin I, Keeler GD, Xiao JW, Basner-Tschakarjan E, High KA, Ertl HC, Rice CM, Srivastava A, de Jong YP, Herzog RW. Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid. Mol Ther. 2016 Jun;24(6):1042-1049. doi: 10.1038/mt.2016.61. Epub 2016 Mar 29. PubMed PMID: 27019999; PubMed Central PMCID: PMC4923326.
  13. Xiao Y, Kwon KC, Hoffman BE, Kamesh A, Jones NT, Herzog RW, Daniell H. Low cost delivery of proteins bioencapsulated in plant cells to human non-immune or immune modulatory cells. Biomaterials. 2016 Feb;80:68-79. doi: 10.1016/j.biomaterials.2015.11.051. Epub 2015 Dec 2. PubMed PMID: 26706477; PubMed Central PMCID: PMC4706487.
  14. Kumar S, Hoffman BE, Herzog R. 79. Vector Dose Delineates Between Chronic, Non-Functional CD8+ T Cell Response and Tolerance to the Transgene Product Upon Liver Gene Transfer. Molecular Therapy. 2016; 24:S35.
  15. Hoffman BE, Keeler G. Vector mediated in vivo induction of antigen-specific regulatory T cells abrogates clinical and histological signs of EAE. The Journal of Immunology. 2016; 196(1 Supplement):139-16.
  16. Pandya M, Britt K, Hoffman BE, Ling C, Aslanidi GV. Reprogramming Immune Response With Capsid-Optimized AAV6 Vectors for Immunotherapy of Cancer. J Immunother. 2015 Sep;38(7):292-8. doi: 10.1097/CJI.0000000000000093. PubMed PMID: 26261893; PubMed Central PMCID: PMC4535186.
  17. Hoffman BE. AAV-based gene therapy for multiple sclerosis. Google Patents; 2015 April. Available from: https://www.google.com/patents/WO2015164789A1?cl=en.
  18. Wang X, Su J, Sherman A, Rogers GL, Liao G, Hoffman BE, Leong KW, Terhorst C, Daniell H, Herzog RW. Plant-based oral tolerance to hemophilia therapy employs a complex immune regulatory response including LAP+CD4+ T cells. Blood. 2015 Apr 9;125(15):2418-27. doi: 10.1182/blood-2014-08-597070. Epub 2015 Feb 19. PubMed PMID: 25700434; PubMed Central PMCID: PMC4392010.
  19. Herzog R, Hoffman BE, Zolotukhin I, High K, Srivastava A, de Jong Y, Vercauteren K, Xiao J. AAV3 Capsid Is Superior for In Vivo Gene Transfer to Human Hepatocytes Compared to Serotypes 5 and 8 in a Mouse/Human Chimeric Model. Blood. 2015; 126(23):4418-4418.
  20. Hoffman BE. 521. AAV Immunotherapy Induces Functional Antigen Specific Regulatory T-Cells to a Neuroantigen: A Potential Treatment for MS. Molecular Therapy. 2015; 23:S209.
  21. Pandya M, Britt K, Ling C, Hoffman BE, Aslanidi G. 221. The Utilization of Capsid-Optimized Adeno-Associated Virus (AAV) Vectors for Cancer Immunotherapy. Molecular Therapy. 2015; 23:1.
  22. Hoffman, Brad E.. AAV Immunotherapy Induces Functional Antigen Specific Regulatory T-Cells to a Neuroantigen: A Potential Treatment for MS. Molecular Therapy. 2015; 23:S209.
  23. Sarkar D, Biswas M, Liao G, Seay HR, Perrin GQ, Markusic DM, Hoffman BE, Brusko TM, Terhorst C, Herzog RW. Ex Vivo Expanded Autologous Polyclonal Regulatory T Cells Suppress Inhibitor Formation in Hemophilia. Mol Ther Methods Clin Dev. 2014 Jul 30;1. doi: 10.1038/mtm.2014.30. PubMed PMID: 25364772; PubMed Central PMCID: PMC4213815.
  24. Palaschak B, Aslanidi G, Cooper M, Hoffman BE. Re-Establishing Immune Tolerance to Neuroantigens by AAV Gene Therapy. MOLECULAR THERAPY. 2014; 22:S304-S304.
  25. Palaschak, Brett, Aslanidi, George V., Cooper, Mario, Hoffman, Brad E.. Re-Establishing Immune Tolerance to Neuroantigens by AAV Gene Therapy. Molecular Therapy. 2014; 22:S304.
  26. Markusic DM, Hoffman BE, Perrin GQ, Nayak S, Wang X, LoDuca PA, High KA, Herzog RW. Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies. EMBO Mol Med. 2013 Nov;5(11):1698-709. doi: 10.1002/emmm.201302859. Epub 2013 Sep 16. PubMed PMID: 24106230; PubMed Central PMCID: PMC3840486.
  27. Hoffman BE, Herzog RW. Covert warfare against the immune system: decoy capsids, stealth genomes, and suppressors. Mol Ther. 2013 Sep;21(9):1648-50. doi: 10.1038/mt.2013.176. PubMed PMID: 24008618; PubMed Central PMCID: PMC3776640.
  28. Zolotukhin I, Luo D, Gorbatyuk O, Hoffman BE, Warrington K Jr, Herzog R, Harrison J, Cao O. Improved Adeno-associated Viral Gene Transfer to Murine Glioma. J Genet Syndr Gene Ther. 2013 Apr 29;4(133). doi: 10.4172/2157-7412.1000133. PubMed PMID: 24319629; PubMed Central PMCID: PMC3852404.
  29. Zolotukhin I, Luo D, Gorbatyuk O, Hoffman BE, Warrington Jr K, Herzog R, Harrison J, Cao O. Improved adeno-associated viral gene transfer to Murine glioma. Journal of genetic syndrome & gene therapy. 2013; 4(133).
  30. Hoffman BE, Zolotukhin I, Herzog R, Cao O. Capsid Modified AAV2 Vectors Are Capable of Generating Functional Cytotoxic T Cells in a Prime-Boost Vaccine Protocol. MOLECULAR THERAPY. 2013; 21:S74-S75.
  31. I Zolotukhin  1 , D Luo, Os Gorbatyuk, Hoffman BE, Kh Warrington Jr, Rw Herzog, Jk Harrison, O Cao. Improved Adeno-associated Viral Gene Transfer to Murine Glioma. Journal of Genetic Syndromes & Gene Therapy. 2013; 04(03):-. doi: 10.4172/2157-7412.1000133.
  32. Hoffman, Brad E., Zolotukhin, Irene, Herzog, Roland W., Cao, Ou. Capsid Modified AAV2 Vectors Are Capable of Generating Functional Cytotoxic T Cells in a Prime-Boost Vaccine Protocol. Molecular Therapy. 2013; 21:S74-S75.
  33. Rogers GL, Hoffman BE. Optimal Immunofluorescent Staining for Human Factor IX and Infiltrating T Cells following Gene Therapy for Hemophilia B. J Genet Syndr Gene Ther. 2012 Aug 15;S1. doi: 10.4172/2157-7412.s1-012. PubMed PMID: 23264888; PubMed Central PMCID: PMC3526188.
  34. Hoffman BE, Ertl HC, Terhorst C, High KA, Herzog RW. Gene therapy research at the frontiers of viral immunology. Front Microbiol. 2012;3:182. doi: 10.3389/fmicb.2012.00182. eCollection 2012. PubMed PMID: 22783235; PubMed Central PMCID: PMC3390768.
  35. Zolotukhin I, Luo D, Hoffman BE, Herzog R, Harrison J, Cao O. Optimization of rAAV for Anti-Glioma Therapy. MOLECULAR THERAPY. 2012; 20:S226-S227.
  36. Rogers G, Hoffman BE. Optimal immunofluorescent staining for human factor IX and infiltrating T cells following gene therapy for hemophilia B. Journal of genetic syndrome & gene therapy. 2012.
  37. Hoffman BE, Zolotukhin I, Herzog R, Cao O. Capsid Modified AAV2 Vectors Are Capable of Generating Vaccine-Mediated Protection. MOLECULAR THERAPY. 2012; 20:S257-S258.
  38. Hoffman BE, Ertl H, Terhorst C, High K, Herzog R. Gene therapy research at the frontiers of viral immunology. Approaches to Blocking the Immune Response to Gene Transfer with Viral Vectors. 2012; :4.
  39. Hoffman, Brad E., Zolotukhin, Irene, Herzog, Roland W., Cao, Ou. Capsid Modified AAV2 Vectors Are Capable of Generating Vaccine-Mediated Protection. Molecular Therapy. 2012; 20:S257-S258.
  40. Zolotukhin, Irene, Luo, Defang, Hoffman, Brad, Herzog, Roland, Harrison, Jeff, Cao, Ou. Optimization of rAAV for Anti-Glioma Therapy. Molecular Therapy. 2012; 20:S226-S227.
  41. Nayak S, Sarkar D, Perrin GQ, Moghimi B, Hoffman BE, Zhou S, Byrne BJ, Herzog RW. Prevention and Reversal of Antibody Responses Against Factor IX in Gene Therapy for Hemophilia B. Front Microbiol. 2011;2:244. doi: 10.3389/fmicb.2011.00244. eCollection 2011. PubMed PMID: 22279442; PubMed Central PMCID: PMC3260742.
  42. Hoffman BE, Martino AT, Sack BK, Cao O, Liao G, Terhorst C, Herzog RW. Nonredundant roles of IL-10 and TGF-β in suppression of immune responses to hepatic AAV-factor IX gene transfer. Mol Ther. 2011 Jul;19(7):1263-72. doi: 10.1038/mt.2011.33. Epub 2011 Mar 8. PubMed PMID: 21386826; PubMed Central PMCID: PMC3129563.
  43. Nayak S, Sarkar D, Perrin G, Moghimi B, Hoffman BE, Zhou S, Byrne B, Herzog R. Prevention and reversal of antibody responses against factor IX in gene therapy for hemophilia B. Approaches to Blocking the Immune Response to Gene Transfer with Viral Vectors. 2011; :65.
  44. Markusic DM, Herzog RW, Aslanidi GV, Hoffman BE, Li B, Li M, Jayandharan GR, Ling C, Zolotukhin I, Ma W, Zolotukhin S, Srivastava A, Zhong L. High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines. Mol Ther. 2010 Dec;18(12):2048-56. doi: 10.1038/mt.2010.172. Epub 2010 Aug 24. PubMed PMID: 20736929; PubMed Central PMCID: PMC2997584.
  45. Verma D, Moghimi B, LoDuca PA, Singh HD, Hoffman BE, Herzog RW, Daniell H. Oral delivery of bioencapsulated coagulation factor IX prevents inhibitor formation and fatal anaphylaxis in hemophilia B mice. Proc Natl Acad Sci U S A. 2010 Apr 13;107(15):7101-6. doi: 10.1073/pnas.0912181107. Epub 2010 Mar 29. PubMed PMID: 20351275; PubMed Central PMCID: PMC2872434.
  46. Verma D, Moghimi B, LoDuca P, Singh H, Hoffman BE, Herzog R, Daniell H. Oral delivery of bioencapsulated coagulation factor IX prevents inhibitor formation and fatal anaphylaxis in hemophilia B mice. Proceedings of the National Academy of Sciences. 2010 March; 107(15):7101-7106. doi: 10.1073/pnas.0912181107.
  47. Verma D, Moghimi B, LoDuca P, Singh H, Hoffman BE, Herzog R, Daniell H. Oral delivery of bioencapsulated coagulation factor IX prevents inhibitor formation and fatal anaphylaxis in hemophilia B mice. Proceedings of the National Academy of Sciences. 2010; 107(15):7101-7106.
  48. Cao O, Hoffman BE, Moghimi B, Nayak S, Cooper M, Zhou S, Ertl HC, High KA, Herzog RW. Impact of the underlying mutation and the route of vector administration on immune responses to factor IX in gene therapy for hemophilia B. Mol Ther. 2009 Oct;17(10):1733-42. doi: 10.1038/mt.2009.159. Epub 2009 Jul 14. PubMed PMID: 19603001; PubMed Central PMCID: PMC2835008.
  49. Nayak S, Cao O, Hoffman BE, Cooper M, Zhou S, Atkinson MA, Herzog RW. Prophylactic immune tolerance induced by changing the ratio of antigen-specific effector to regulatory T cells. J Thromb Haemost. 2009 Sep;7(9):1523-32. doi: 10.1111/j.1538-7836.2009.03548.x. Epub 2009 Jul 6. PubMed PMID: 19583824; PubMed Central PMCID: PMC2981868.
  50. Martino AT, Nayak S, Hoffman BE, Cooper M, Liao G, Markusic DM, Byrne BJ, Terhorst C, Herzog RW. Tolerance induction to cytoplasmic beta-galactosidase by hepatic AAV gene transfer: implications for antigen presentation and immunotoxicity. PLoS One. 2009 Aug 4;4(8):e6376. doi: 10.1371/journal.pone.0006376. PubMed PMID: 19652717; PubMed Central PMCID: PMC2715858.
  51. Cooper M, Nayak S, Hoffman BE, Terhorst C, Cao O, Herzog RW. Improved induction of immune tolerance to factor IX by hepatic AAV-8 gene transfer. Hum Gene Ther. 2009 Jul;20(7):767-76. doi: 10.1089/hum.2008.161. PubMed PMID: 19309290; PubMed Central PMCID: PMC2829279.
  52. LoDuca PA, Hoffman BE, Herzog RW. Hepatic gene transfer as a means of tolerance induction to transgene products. Curr Gene Ther. 2009 Apr;9(2):104-14. doi: 10.2174/156652309787909490. Review. PubMed PMID: 19355868; PubMed Central PMCID: PMC2851180.
  53. Martino A, Nayak S, Hoffman BE, Cooper M, Liao G, Giannobile W. Tolerance Induction to Cytoplasmic b-Galactosidase by Hepatic AAV Gene Transfer—. 2009.
  54. Martino A, Nayak S, Cooper M, Hoffman BE, Campbell-Thompson M, Herzog R. Systemic Expression of Transgene Product Is Not Required for Induction of Immune Tolerance by AAV2 Hepatic Gene Transfer. MOLECULAR THERAPY. 2009; 17:S379-S379.
  55. Hoffman BE, Cao O, Moghimi B, Nayak S, Cooper M, Zhou S, Ertl H, High K, Herzog R. Substantial impact of the genetic mutation and the gene transfer protocol on immune responses to factor Ix in Aav-based therapy for hemophilia B. Journal of Thrombosis and Haemostasis. 2009; 7:35.
  56. Martino, AT, Nayak, S, Cooper, M, Hoffman, BE, Campbell-Thompson, M, Herzog, RW. Systemic Expression of Transgene Product Is Not Required for Induction of Immune Tolerance by AAV2 Hepatic Gene Transfer. Molecular Therapy. 2009; 17:S379.
  57. Hoffman BE, Herzog RW. Coaxing the liver into preventing autoimmune disease in the brain. J Clin Invest. 2008 Oct;118(10):3271-3. doi: 10.1172/JCI37079. PubMed PMID: 18802483; PubMed Central PMCID: PMC2542853.
  58. Hoffman BE, Herzog RW. Immunology of Hepatic Gene Therapy. In: Herzog RW, editor. Gene Therapy: Immunology USA: Wiley-Blackwell; 2008. Chapter 8;
  59. Nayak S, Hoffman BE, Cooper M, Atkinson M, Cao O, Herzog R. A Prophylactic Protocol for the Prevention of Inhibitor Formation in Gene Therapy for Hemophilia B by Shifting the Balance from An Effector to a Regulatory T Cell Response. Blood. 2008; 112(11):3531-3531.
  60. Hoffman, BE, Herzog, RW. Immunology of hepatic gene transfer. In: Herzog, RW, editor. Immunology of Gene Therapy 2008. Chapter Chapter 8.
  61. Hoffman BE, Dobrzynski E, Wang L, Hirao L, Mingozzi F, Cao O, Herzog RW. Muscle as a target for supplementary factor IX gene transfer. Hum Gene Ther. 2007 Jul;18(7):603-13. doi: 10.1089/hum.2007.042. PubMed PMID: 17594244.
  62. Hoffman BE, Herzog R. Initial Identification of a Mouse Human Factor IX-Specific CD8+ T-Cell Epitope. Blood. 2006; 108(11):5490-5490.
  63. Hoffman BE, Chang J, Katsetos C, Platsoucas C, Oleszak E. T cells infiltrating the CNS of Theiler’s virus (TMEV)-infected mice during late chronic demyelinating disease are G0/G1 arrested and do not undergo apoptosis. JOURNAL OF NEUROIMMUNOLOGY. 2004; 154(1-2):204-204.
  64. Hoffman, BE, Chang, JR, Katsetos, CD, Platsoucas, CD, Oleszak, EL. T cells infiltrating the CNS of Theiler’s virus (TMEV)-infected mice during late chronic demyelinating disease are G0/G1 arrested and do not undergo apoptosis. Journal of Neuroimmunology. 2004; 154(1-2):204.
  65. Oleszak EL, Hoffman BE, Chang JR, Zaczynska E, Gaughan J, Katsetos CD, Platsoucas CD, Harvey N. Apoptosis of infiltrating T cells in the central nervous system of mice infected with Theiler’s murine encephalomyelitis virus. Virology. 2003 Oct 10;315(1):110-23. doi: 10.1016/s0042-6822(03)00517-8. PubMed PMID: 14592764.
  66. Prineas JW, Kwon EE, Cho ES, Sharer LR, Barnett MH, Oleszak EL, Hoffman BE, Morgan BP. Immunopathology of secondary-progressive multiple sclerosis. Ann Neurol. 2001 Nov;50(5):646-57. doi: 10.1002/ana.1255. PubMed PMID: 11706971.
  67. Oleszak EL, Lin WL, Legido A, Melvin J, Hardison H, Hoffman BE, Katsetos CD, Platsoucas CD. Presence of oligoclonal T cells in cerebrospinal fluid of a child with multiphasic disseminated encephalomyelitis following hepatitis A virus infection. Clin Diagn Lab Immunol. 2001 Sep;8(5):984-92. doi: 10.1128/CDLI.8.5.984-992.2001. PubMed PMID: 11527815; PubMed Central PMCID: PMC96183.
  68. Oleszak E, Lin W, Legido A, Melvin J, Hardison H, Hoffman BE, Katsetos C, Platsoucas C. Presence of Oligoclonal T Cells in Cerebrospinal Fluid of a Child with Multiphasic Disseminated Encephalomyelitis following Hepatitis A Virus Infection. Clinical and Vaccine Immunology. 2001 September; 8(5):984-992. doi: 10.1128/CDLI.8.5.984-992.2001.
  69. Oleszak E, Lin W, Legido A, Melvin J, Hardison H, Hoffman BE, Katsetos C, Platsoucas C. IMMUNE-MEDIATED RESPONSES AND DISORDERS-Presence of Oligoclonal T Cells in Cerebrospinal Fluid of a Child with Multiphasic Disseminated Encephalomyelitis following Hepatitis A Virus Infection. Clinical and Diagnostic Laboratory Immunology. 2001; 8(5):984-992.
  70. Hoffman BE, Katsetos C, Zaczynska E, Harvey N, Oleszak E. Apoptosis in the CNS of they-infected SJL mice. FASEB JOURNAL. 1998; 12(5):A798-A798.