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.

Abstract

In the current studies we generated novel capsid-optimized adeno-associated virus (AAV) serotype 6 (AAV6) vectors expressing a tumor-associated antigen, and assessed their ability to activate a protective T-cell response in an animal model. First, we showed that specific mutations in the AAV6 capsid increase the transduction efficiency of these vectors in mouse bone marrow-derived dendritic cells in vitro for approximately 5-fold compared with the wild-type (WT) AAV6 vectors. Next, we evaluated the ability of the mutant AAV6 vectors to initiate specific T-cell clone proliferation in vivo. Our data indicate that the intramuscular administration of AAV6-S663V+T492V vectors expressing ovalbumin (OVA) led to a strong activation (approximately 9%) of specific T cells in peripheral blood compared with AAV6-WT treated animals (<1%). These OVA-specific T cells have a superior killing ability against mouse prostate cancer cell line RM1 stably expressing the OVA antigen when propagated in vitro. Finally, we evaluated the ability of capsid-optimized AAV6-S663V+T492V vectors to initiate a protective anticancer immune response in vivo. Our results document the suppression of subcutaneous tumor growth in animals immunized with AAV6-S663V+T492V vectors expressing prostatic acid phosphatase (PAP) for approximately 4 weeks in comparison with 1 week and 2 weeks for the negative controls, AAV6-EGFP, and AAV6-WT-PAP treated mice, respectively. These studies suggest that successful inhibition of tumor growth in an animal model would set the stage for potential clinical application of the capsid-optimized AAV6-S663V+T492V vectors.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Capsid / immunology*
  • Capsid Proteins / immunology*
  • Cell Line, Tumor
  • Dependovirus / immunology*
  • Genetic Therapy / methods
  • Genetic Vectors / immunology*
  • Immunotherapy / methods
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Neoplasms / immunology*
  • Ovalbumin / immunology
  • Prostatic Neoplasms / immunology
  • T-Lymphocytes / immunology
  • Transduction, Genetic / methods

Substances

  • Capsid Proteins
  • Ovalbumin