Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers

J Virol. 1998 May;72(5):4212-23. doi: 10.1128/JVI.72.5.4212-4223.1998.

Abstract

Immune responses to vector-corrected cells have limited the application of gene therapy for treatment of chronic disorders such as inherited deficiency states. We have found that recombinant adeno-associated virus (AAV) efficiently transduces muscle fibers in vivo without activation of cellular and humoral immunity to neoantigenic transgene products such as beta-galactosidase, which differs from the experience with recombinant adenovirus, where vibrant T-cell responses to the transgene product destroy the targeted muscle fibers. T cells activated following intramuscular administration of adenovirus expressing lacZ (AdlacZ) can destroy AAVlacZ-transduced muscle fibers, indicating a prior state of immunologic nonresponsiveness in the context of AAV gene therapy. Adoptive transfer of dendritic cells infected with AdlacZ leads to immune mediated elimination of AAVlacZ-transduced muscle fibers. AAVlacZ-transduced antigen-presenting cells fail to demonstrate beta-galactosidase activity and are unable to elicit transgene immunity in adoptive transfer experiments. These studies indicate that vector-mediated transduction of dendritic cells is necessary for cellular immune responses to muscle gene therapy, a step which AAV avoids, providing a useful biological niche for its use in gene therapy.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Animals
  • Antigen Presentation
  • CD4-Positive T-Lymphocytes / immunology
  • Cell Transformation, Viral
  • Dendritic Cells / immunology*
  • Dependovirus / immunology*
  • Genes, Reporter
  • Genetic Vectors / immunology*
  • Lymphocyte Activation / immunology
  • Mice
  • Mice, Inbred C57BL
  • Muscle Fibers, Skeletal / immunology*
  • T-Lymphocytes, Cytotoxic / immunology
  • Transgenes
  • beta-Galactosidase / genetics
  • beta-Galactosidase / immunology

Substances

  • beta-Galactosidase