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Related Experiment Videos

Gene therapy in autoimmune disease.

I H Tarner1, C G Fathman

  • 1Stanford University School of Medicine, Department of Medicine, Division of Immunology and Rheumatology, CCSR Building, Stanford, CA 94305-5166, USA. ingo.tarner@stanford.edu

Current Opinion in Immunology
|October 26, 2001
PubMed
Summary

Gene therapy shows promise for autoimmune diseases, using modified cells for targeted treatment. A phase I clinical trial in rheumatoid arthritis patients assessed the safety and feasibility of this innovative gene transfer approach.

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Area of Science:

  • Immunology
  • Gene Therapy
  • Autoimmune Diseases

Background:

  • Autoimmune diseases involve the immune system attacking the body's own tissues.
  • Gene therapy offers a potential strategy to modulate immune responses in autoimmunity.
  • Dendritic cells and T cells are explored as vectors for targeted gene delivery.

Purpose of the Study:

  • To investigate the pathogenesis of autoimmunity through gene therapy research.
  • To evaluate the efficacy and safety of using transduced dendritic cells and antigen-specific T cells for gene therapy.
  • To assess the feasibility and safety of gene transfer in a human clinical trial for rheumatoid arthritis.

Main Methods:

  • Utilizing transduced dendritic cells and antigen-specific T cells as gene delivery vehicles.

Related Experiment Videos

  • Employing bioluminescence imaging to track cell trafficking and homing in vivo.
  • Conducting a phase I clinical trial to evaluate gene transfer in rheumatoid arthritis patients.

    • Main Results:

    • Gene therapy applications using specific cell types have demonstrated reliability and effectiveness.
    • Bioluminescence imaging successfully visualized in vivo cell trafficking and homing.
    • A phase I clinical trial in rheumatoid arthritis patients was completed, providing initial safety and feasibility data.

    Conclusions:

    • Gene therapy, particularly with engineered immune cells, offers a promising avenue for treating autoimmune diseases.
    • In vivo imaging techniques are valuable for understanding cell-based therapy dynamics.
    • The completed phase I trial represents a crucial first step towards human application of gene therapy for rheumatoid arthritis.