Research Topics



Affiliation: University of Florida
Country: USA


  1. Perrin G, Herzog R, Markusic D. Update on clinical gene therapy for hemophilia. Blood. 2018;: pubmed publisher
    ..The current review provides a summary and update on advances in clinical gene therapies for hemophilia and its continued development. ..
  2. Herzog R, Cao O, Srivastava A. Two decades of clinical gene therapy--success is finally mounting. Discov Med. 2010;9:105-11 pubmed
    ..While immune responses and insertional mutagenesis pose obstacles for this novel form of molecular medicine, continuous progress suggests that a wider range of diseases can be treated with gene therapy in the future. ..
  3. Su J, Zhu L, Sherman A, Wang X, Lin S, Kamesh A, et al. Low cost industrial production of coagulation factor IX bioencapsulated in lettuce cells for oral tolerance induction in hemophilia B. Biomaterials. 2015;70:84-93 pubmed publisher
  4. Wang X, Terhorst C, Herzog R. In vivo induction of regulatory T cells for immune tolerance in hemophilia. Cell Immunol. 2016;301:18-29 pubmed publisher
    ..Among others, drug- and gene transfer-based protocols, lymphocyte transplant, and oral tolerance are reviewed. ..
  5. request reprint
    Herzog R. Immune responses to AAV capsid: are mice not humans after all?. Mol Ther. 2007;15:649-50 pubmed
  6. Biswas M, Rogers G, Sherman A, Byrne B, Markusic D, Jiang H, et al. Combination therapy for inhibitor reversal in haemophilia A using monoclonal anti-CD20 and rapamycin. Thromb Haemost. 2017;117:33-43 pubmed publisher
    ..This regimen represents a promising treatment for inhibitor reversal in HA, as both of these compounds have been extensively used in human patients. ..
  7. Vercauteren K, Hoffman B, Zolotukhin I, Keeler G, Xiao J, Basner Tschakarjan E, et al. Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid. Mol Ther. 2016;24:1042-1049 pubmed publisher
  8. Kumar S, Hoffman B, Terhorst C, De Jong Y, Herzog R. 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;25:880-891 pubmed publisher
    ..Regulatory T cells, IL-10 expression, and Fas-L contributed to high-dose tolerance. Thus, viral vector doses profoundly impact CD8+ T cell responses. ..
  9. Markusic D, Nichols T, Merricks E, Palaschak B, Zolotukhin I, Marsic D, et al. Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models. J Transl Med. 2017;15:94 pubmed publisher
    ..However, capsid optimization solely in mouse liver may not predict efficacy in other species and thus is of limited translational utility. ..

More Information


  1. Herzog R. Complexity of immune responses to AAV transgene products - Example of factor IX. Cell Immunol. 2017;: pubmed publisher
    ..Furthermore, the potential for tolerance induction by hepatic gene transfer or combination with immune modulation is discussed. ..
  2. Herzog R, Cooper M, Perrin G, Biswas M, Martino A, Morel L, et al. Regulatory T cells and TLR9 activation shape antibody formation to a secreted transgene product in AAV muscle gene transfer. Cell Immunol. 2017;: pubmed publisher
    ..Further, Treg re-population eventually resolved humoral and cellular immune responses. Therefore, specific modes of TLR9 activation and Tregs orchestrate antibody formation in muscle gene transfer. ..
  3. Corti M, Liberati C, Smith B, Lawson L, Tuna I, Conlon T, et al. Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in children affected by Pompe Disease. Hum Gene Ther Clin Dev. 2017;: pubmed publisher
    ..This observation is significant for future gene therapy studies and serves to establish a clinically relevant approach to blocking immune responses to both the AAV capsid protein and transgene product...