Brad E Hoffman

Brad E Hoffman, PhD

Associate Professor

Business Phone: (352) 273-8152
Business Email:

About Brad E Hoffman

Associate Professor,

Department of Pediatrics

Department of Neuroscience

Research Profile

The major focus of my research program is aimed at understanding and exploiting the unique molecular and cellular mechanisms required for the induction of immunological tolerance. As such, my research group is developing novel immunotherapies using the Adeno-associated virus (AAV) gene-therapy platform that are capable of inducing tolerogenic antigen-specific regulatory T cells (Tregs) as a therapeutic intervention for autoimmune and genetic disease such as Multiple Sclerosis. Recently, we were the first to demonstrate that an AAV vector could be used therapeutically to not only prevent development of disease, but to also abrogate preexisting (reverse) disease in multiple mouse models of Multiple Sclerosis.

Open Researcher and Contributor ID (ORCID)


Areas of Interest
  • Adeno-Associated Viral Gene Therapy
  • Autoimmune Disease
  • Gene therapy
  • Immune Tolerance
  • Immunotherapy
  • Multiple Sclerosis


PhD Immunology
2006 · Temple University School of Medicine
BS Chemistry & Biology
1994 · University of Central Florida


Research Scientist Award
2018 · National multiple sclerosis society
2017-current · NIH-LRP Program
Recipient, Travel Grant
2016 · International Congress of Immunology
Recipient, Travel Grant
2016 · American Association of Immunologists
Mentor S.T.E.M Supernova
2015-current · Boy Scouts of America
Award in Hemophilia Research (ASPIRE)
2011 · Pfizer Pharmaceuticals
Immunology Grant Recipient
2011 · BD Bioscience
Kokomoor Award for Excellence in Pediatric Research
2010 · University of Florida, Department of Pediatrics
Research Fellowship
2007 · American Heart Association


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 therapy. 28(7-8):422-434 [DOI] 10.1038/s41434-020-0140-1. [PMID] 32152434.
Enhanced Transduction of Human Hematopoietic Stem Cells by AAV6 Vectors: Implications in Gene Therapy and Genome Editing.
Molecular therapy. Nucleic acids. 20:451-458 [DOI] 10.1016/j.omtn.2020.03.009. [PMID] 32276210.
Type I IFN Sensing by cDCs and CD4+ T Cell Help Are Both Requisite for Cross-Priming of AAV Capsid-Specific CD8+ T Cells.
Molecular therapy : the journal of the American Society of Gene Therapy. 28(3):758-770 [DOI] 10.1016/j.ymthe.2019.11.011. [PMID] 31780366.
Liver induced transgene tolerance with AAV vectors.
Cellular immunology. 342 [DOI] 10.1016/j.cellimm.2017.12.002. [PMID] 29576315.
Regulatory T cells and TLR9 activation shape antibody formation to a secreted transgene product in AAV muscle gene transfer.
Cellular immunology. 342 [DOI] 10.1016/j.cellimm.2017.07.012. [PMID] 28888664.
Gene Therapy-Induced Antigen-Specific Tregs Inhibit Neuro-inflammation and Reverse Disease in a Mouse Model of Multiple Sclerosis.
Molecular therapy : the journal of the American Society of Gene Therapy. 26(1):173-183 [DOI] 10.1016/j.ymthe.2017.09.001. [PMID] 28943274.
Alpha-1 Antitrypsin-Deficient Macrophages Have Increased Matriptase-Mediated Proteolytic Activity.
American journal of respiratory cell and molecular biology. 57(2):238-247 [DOI] 10.1165/rcmb.2016-0366OC. [PMID] 28362108.
Plasmacytoid and conventional dendritic cells cooperate in crosspriming AAV capsid-specific CD8+ T cells.
Blood. 129(24):3184-3195 [DOI] 10.1182/blood-2016-11-751040. [PMID] 28468798.
The Balance between CD8+ T Cell-Mediated Clearance of AAV-Encoded Antigen in the Liver and Tolerance Is Dependent on the Vector Dose.
Molecular therapy : the journal of the American Society of Gene Therapy. 25(4):880-891 [DOI] 10.1016/j.ymthe.2017.02.014. [PMID] 28284982.
Low cost delivery of proteins bioencapsulated in plant cells to human non-immune or immune modulatory cells.
Biomaterials. 80:68-79 [DOI] 10.1016/j.biomaterials.2015.11.051. [PMID] 26706477.
Potential for cellular stress response to hepatic factor VIII expression from AAV vector.
Molecular therapy. Methods & clinical development. 3 [PMID] 27738644.
View on: PubMed
Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid.
Molecular therapy : the journal of the American Society of Gene Therapy. 24(6):1042-1049 [DOI] 10.1038/mt.2016.61. [PMID] 27019999.
Plant-based oral tolerance to hemophilia therapy employs a complex immune regulatory response including LAP+CD4+ T cells.
Blood. 125(15):2418-27 [DOI] 10.1182/blood-2014-08-597070. [PMID] 25700434.
Reprogramming Immune Response With Capsid-Optimized AAV6 Vectors for Immunotherapy of Cancer.
Journal of immunotherapy (Hagerstown, Md. : 1997). 38(7):292-8 [DOI] 10.1097/CJI.0000000000000093. [PMID] 26261893.
Ex Vivo Expanded Autologous Polyclonal Regulatory T Cells Suppress Inhibitor Formation in Hemophilia.
Molecular therapy. Methods & clinical development. 1 [PMID] 25364772.
View on: PubMed
Covert warfare against the immune system: decoy capsids, stealth genomes, and suppressors.
Molecular therapy : the journal of the American Society of Gene Therapy. 21(9):1648-50 [DOI] 10.1038/mt.2013.176. [PMID] 24008618.
Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies.
EMBO molecular medicine. 5(11):1698-709 [DOI] 10.1002/emmm.201302859. [PMID] 24106230.
Capsid Modified Aav2 Vectors Are Capable of Generating Vaccine-Mediated Protection
Molecular Therapy. 20:S257-S258
Gene therapy research at the frontiers of viral immunology.
Frontiers in microbiology. 3 [DOI] 10.3389/fmicb.2012.00182. [PMID] 22783235.
Optimal Immunofluorescent Staining for Human Factor IX and Infiltrating T Cells following Gene Therapy for Hemophilia B.
Journal of genetic syndromes & gene therapy. S1 [PMID] 23264888.
View on: PubMed
Optimization of Raav for Anti-Glioma Therapy
Molecular Therapy. 20:S226-S227
Nonredundant roles of IL-10 and TGF-β in suppression of immune responses to hepatic AAV-factor IX gene transfer.
Molecular therapy : the journal of the American Society of Gene Therapy. 19(7):1263-72 [DOI] 10.1038/mt.2011.33. [PMID] 21386826.
Prevention and Reversal of Antibody Responses Against Factor IX in Gene Therapy for Hemophilia B.
Frontiers in microbiology. 2 [DOI] 10.3389/fmicb.2011.00244. [PMID] 22279442.
High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines.
Molecular therapy : the journal of the American Society of Gene Therapy. 18(12):2048-56 [DOI] 10.1038/mt.2010.172. [PMID] 20736929.
Oral delivery of bioencapsulated coagulation factor IX prevents inhibitor formation and fatal anaphylaxis in hemophilia B mice.
Proceedings of the National Academy of Sciences of the United States of America. 107(15):7101-6 [DOI] 10.1073/pnas.0912181107. [PMID] 20351275.
Hepatic Gene Transfer as a Means of Tolerance Induction To Transgene Products
. 9:104-114
Hepatic gene transfer as a means of tolerance induction to transgene products.
Current gene therapy. 9(2):104-14 [PMID] 19355868.
View on: PubMed
Impact of the underlying mutation and the route of vector administration on immune responses to factor IX in gene therapy for hemophilia B.
Molecular therapy : the journal of the American Society of Gene Therapy. 17(10):1733-42 [DOI] 10.1038/mt.2009.159. [PMID] 19603001.
Improved induction of immune tolerance to factor IX by hepatic AAV-8 gene transfer.
Human gene therapy. 20(7):767-76 [DOI] 10.1089/hum.2008.161. [PMID] 19309290.
Prophylactic immune tolerance induced by changing the ratio of antigen-specific effector to regulatory T cells.
Journal of thrombosis and haemostasis : JTH. 7(9):1523-32 [DOI] 10.1111/j.1538-7836.2009.03548.x. [PMID] 19583824.
Systemic Expression of Transgene Product Is Not Required for Induction of Immune Tolerance By Aav2 Hepatic Gene Transfer
Molecular Therapy. 17
Tolerance induction to cytoplasmic beta-galactosidase by hepatic AAV gene transfer: implications for antigen presentation and immunotoxicity.
PloS one. 4(8) [DOI] 10.1371/journal.pone.0006376. [PMID] 19652717.
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. 112
Coaxing the liver into preventing autoimmune disease in the brain.
The Journal of clinical investigation. 118(10):3271-3 [DOI] 10.1172/JCI37079. [PMID] 18802483.
Muscle as a target for supplementary factor IX gene transfer.
Human gene therapy. 18(7):603-13 [PMID] 17594244.
View on: PubMed
Apoptosis of infiltrating T cells in the central nervous system of mice infected with Theiler’s murine encephalomyelitis virus.
Virology. 315(1):110-23 [PMID] 14592764.
View on: PubMed
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. 8(5):984-92 [PMID] 11527815.
View on: PubMed


Published April 2015
AAV-Based Gene Therapy for Multiple Sclerosis

Teaching Profile

Courses Taught
GMS6253 Molecular Therapy III ? Immunology of Gene Transfer
GMS6034 Advanced Virology I: Genetics and RNA
GMS6024 Princ Neuroscience 4

Contact Details

(352) 273-8152
Administrative Assistant:
Cristina D Gaddie
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