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Related Experiment Videos

Gene therapy in transplantation.

D Chen1, R Sung, J S Bromberg

  • 1Carl C. Icahn Institute for Gene Therapy and Molecular Medicine and the Recanati/Miller Transplantation Institute, Mount Sinai School of Medicine, New York, NY 10029-6574, USA.

Transplant Immunology
|August 16, 2002
PubMed
Summary

Gene transfer and gene therapy offer promising avenues for transplantation, targeting immune responses and graft protection. Further research is crucial to overcome barriers and advance these gene medicines into clinical practice.

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Area of Science:

  • Immunology
  • Molecular Biology
  • Transplantation Science

Background:

  • Gene transfer and gene therapy are rapidly advancing fields with emerging applications in transplantation.
  • Current use of gene medicines in transplantation is in its early stages, requiring significant development.

Purpose of the Study:

  • To explore the potential of gene transfer and gene therapy in transplantation.
  • To identify immunologic and non-immunologic targets for gene delivery to grafts.
  • To examine strategies for immune system modification to induce tolerance.

Main Methods:

  • Delivery of vectors to the graft to target immunologic molecules (e.g., costimulatory signals, cytokines) and non-immunologic pathways (e.g., reducing ischemic damage).
  • Delivery of vectors to the immune system to induce tolerance.

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  • Exploration of advanced technologies like gene conversion and xenotransplantation for genetically modified organs.

    • Main Results:

    • Gene transfer can target graft-specific immunologic and non-immunologic pathways to enhance protection and reduce damage.
    • Vector delivery to the immune system holds potential for inducing transplant tolerance.
    • Regulation of transgene expression, vector components, and immune responses are critical factors.

    Conclusions:

    • Gene transfer presents significant opportunities to improve transplantation outcomes by modulating immune responses and protecting grafts.
    • Addressing challenges related to immune responses to vectors and transgene products, and understanding promoter interactions, are key for clinical translation.
    • Continued basic and translational research is essential to overcome barriers and realize the clinical potential of gene medicines in transplantation.