Type 1 Diabetes Mellitus: Immune Modulation as a Prerequisite for Successful Gene Therapy Strategies

Wenhao Chen, Aini Xie, Jie Wu, Lawrence Chan

Research output: Chapter in Book/Report/Conference proceedingChapter


Type 1 diabetes (T1D) is an autoimmune disease for which there is no cure. Pancreatic beta cells are the source of insulin that keeps blood glucose normal. When susceptible individuals develop T1D, their beta cells are destroyed by autoimmune T lymphocytes and no longer produce insulin. T1D patients therefore depend on daily insulin injections for survival. Gene therapy in T1D aims at the induction of new islets to replace those that have been destroyed by autoimmunity. A major goal of T1D research is to restore functional beta cell mass while eliminating diabetogenic T cells in the hope of achieving insulin independence. Multiple therapeutic strategies for the generation of new beta cells have been under intense investigations. However, newly formed beta cells would be immediately destroyed by diabetogenic T cells. Therefore, successful islet induction therapy must be supported by potent immunotherapy that will protect the newly formed beta cells. Herein, we will summarize the current information on immunotherapies that aim at modifying T cell response to beta cells. We first outline the immune mechanisms that underlie T1D development and progression and review the scientific background and rationale for specific modes of immunotherapy. Numerous clinical trials using antigen-specific strategies and immune-modifying drugs have been published, though most have proved too toxic or have failed to provide long-term beta cell protection. To develop an effective immunotherapy, there must be a continued effort to define the molecular basis that underlies immune-mediated killing of pancreatic islet cells in T1D.

Original languageEnglish (US)
Title of host publicationTranslating Gene Therapy to the Clinic
Subtitle of host publicationTechniques and Approaches
Number of pages15
ISBN (Electronic)9780128005644
ISBN (Print)9780128005637
StatePublished - 2015


  • Cytokine
  • Gene therapy
  • Immunotherapy
  • T cell
  • Type 1 diabetes

ASJC Scopus subject areas

  • Medicine(all)
  • Biochemistry, Genetics and Molecular Biology(all)


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