Roland W. Herzog, Ph.D.
Cancer and Genetics Research Complex, Rm 203
Cancer and Genetics Research Complex, Rm 230G
My research program seeks to develop a gene therapy for the X-linked bleeding disorder hemophilia and to establish prophylactic immune tolerance protocols for genetic diseases. Severe hemophilia is characterized by frequent spontaneous bleeding. Our work in canine hemophilia has shown that gene transfer can permanently correct this defect, and clinical trials have been ongoing to translate these successes to a therapy for humans. Treatment is only successful of the functional protein expressed by the donated gene is not rejected by the immune system. Using interdisciplinary approaches, my lab is pursuing several alternative strategies to induce tolerance to therapeutic proteins such as hepatic gene transfer, mucosal tolerance, and immune modulation. Important mechanistic aspects of these studies include organ-specific immunity, innate immunity, and immune regulation/regulatory T cells. We are using small and large models of hemophilia to advance basic science toward clinical application. Collaborators in the Department of Pediatrics include Drs. Byrne, Cao, Srivastava, and Zolotukhin.
Dr. Herzog received a Ph.D. in Microbiology from Auburn University in 1996. He subsequently performed postdoctoral research in gene therapy at the Children’s Hospital of Philadelphia, and became Assistant Professor of Pediatrics at the University of Pennsylvania in 2000. In 2005, he joined the University of Florida as an Associate Professor. Dr. Herzog’s research has received multiple awards, including a plenary presentation at the annual meeting of the American Society of Hematology (1998), a career development award from the National Hemophilia Foundation (2000), an outstanding new investigator award from the American Society of Gene and Cell Therapy (ASGCT, 2003), and a Bayer Hemophilia Award (2007). He served as chair of the ASGCT immunology and education committees and edited two textbooks in gene therapy. He is currently Deputy Editor of the journal Molecular Therapy. His laboratory has received multiple grants from the NIH and other funding agencies. In 2011, Dr. Herzog was promoted to Professor and became member of the NIH study section Hemostasis and Thrombosis.
Additional publications can be found in PubMed.
- Mingozzi F, Liu YL, Dobrzynski E, Kaufhold A, Liu JH, Wang YQ, Arruda VR, High KA, Herzog RW (2003) Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. J. Clin. Inv. 111: 1347-1356
- Dobrzynski E, Fitzgerald JC, Cao O, Mingozzi F, Wang L, Herzog RW (2006) Prevention of cytotoxic T lymphocyte responses to factor IX expressing hepatocytes by gene transfer-induced regulatory T cells. Proc Natl Acad Sci USA 103: 4592-4597
- Cao O, Dobrzynski E, Wang L, Nayak S, Mingle B, Terhorst CP, Herzog RW (2007) Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer. Blood 110: 1132-1140
- Verma D, Moghimi B, LoDuca PA, Singh HD, Hoffman BE, Herzog RW, Daniell H (2010) Oral administration of bioencapsulated factor IX prevents inhibitor formation and fatal anaphylaxis in hemophilia B mice. Proc Natl Acad Sci USA 107: 7101-7106
- Martino AT, Suzuki M, Markusic DM, Zolotukhin I, Ryals RC, Moghimi B, Ertl HCJ, Muruve DA, Lee B, Herzog RW (2011) The genome of self-complementary AAV vectors increases TLR9-dependent innate immune responses in the liver. Blood 117: 6459-6468
- Hoffman BE, Martino AT, Sack BK, Cao O, Liao G, Terhorst C, Herzog RW (2011) Non-redundant roles of IL-10 and TGF-β in suppression of immune responses to hepatic AAV-factor IX gene transfer. Mol Ther 19: 1263-1272