Engineering antigen-specific T cells from genetically modified human hematopoietic stem cells in immunodeficient mice

PLoS One. 2009 Dec 7;4(12):e8208. doi: 10.1371/journal.pone.0008208.

Abstract

There is a desperate need for effective therapies to fight chronic viral infections. The immune response is normally fastidious at controlling the majority of viral infections and a therapeutic strategy aimed at reestablishing immune control represents a potentially powerful approach towards treating persistent viral infections. We examined the potential of genetically programming human hematopoietic stem cells to generate mature CD8+ cytotoxic T lymphocytes that express a molecularly cloned, "transgenic" human anti-HIV T cell receptor (TCR). Anti-HIV TCR transduction of human hematopoietic stem cells directed the maturation of a large population of polyfunctional, HIV-specific CD8+ cells capable of recognizing and killing viral antigen-presenting cells. Thus, through this proof-of-concept we propose that genetic engineering of human hematopoietic stem cells will allow the tailoring of effector T cell responses to fight HIV infection or other diseases that are characterized by the loss of immune control.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antigens, Viral / immunology*
  • Antiviral Agents / metabolism
  • Cell Differentiation / immunology
  • Cloning, Molecular
  • Epitopes / immunology*
  • Genetic Engineering*
  • HIV / immunology
  • Hematopoietic Stem Cells / immunology*
  • Humans
  • Mice
  • Mice, SCID
  • Phenotype
  • Receptors, Antigen, T-Cell / immunology
  • Receptors, Antigen, T-Cell / isolation & purification
  • Species Specificity
  • T-Lymphocytes / immunology*
  • T-Lymphocytes / virology

Substances

  • Antigens, Viral
  • Antiviral Agents
  • Epitopes
  • Receptors, Antigen, T-Cell