Herzog Lab
Principal Investigator: Roland W. Herzog
Institution: Indiana University Indianapolis (Indianapolis, IN)
Primary Subfield: Molecular & Cell Bio
Recruitment Status: Inquire for rotation/fellowship opportunities
Research Focus
In recent years, 3 gene therapy protocols for the treatment of the bleeding disorders hemophilia A (coagulation factor FVIII deficiency) and hemophilia B (FIX deficiency) have received regulatory approval, all using in vivo adeno-associated viral (AAV) gene transfer into hepatocytes. We were the first to show that this approach induces regulatory T cells (Treg) and promotes immune tolerance to the transgene product, a phenomenon that has since been replicated in many different animal models. First evidence that this concept applies to humans has now been obtained in a hemophilia A patient whose antibody against FVIII was eliminated by AAV gene transfer. However, we found that the liver is also capable of priming CD8+ T cell responses against the transgene product (through forming dendric cell (DC) - Kupffer cell (KC) - T cell networks). We uncovered critical innate signals and mechanisms that lead to T cell activation, defined a role for levels of gene expression, and developed adjunct immune modulation protocols that further improve immune regulation, reduce responses to viral capsid, and enhance safety of therapy. Here, we will continue combining our team’s expertise in gene tran…
Active NIH Awards
- 5P01HL160472-05: Toward Safer Gene Therapy for Hemophilia A (2026)
- 5R01HL131093-10: Enhancing immune regulation in gene therapy for hemophilia (2026)
- 5R01AI177600-04: Mechanism of immune response to muscle-directed AAV gene transfer (2026)
- 5P01HL160472-05: Mechanisms of Innate and Adaptive Immune Responses to AAV-FVIII Gene Transfer (2026)
- 5P01HL160472-05: Administrative Core (2026)
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