Gene therapy progress and prospects: gene therapy for diabetes mellitus

Gene Ther. 2005 Jan;12(2):101-7. doi: 10.1038/


Diabetes mellitus has long been targeted, as yet unsuccessfully, as being curable with gene therapy. The main hurdles have not only been vector-related toxicity but also the lack of physiological regulation of the expressed insulin. Recent advances in understanding the developmental biology of beta-cells and the transcriptional cascade that drives it have enabled both in vivo and ex vivo gene therapy combined with cell therapy to be used in animal models of diabetes with success. The associated developments in the stem cell biology and immunology have opened up further opportunities for gene therapy to be applied to target autoimmune diabetes.

Publication types

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

MeSH terms

  • Animals
  • Autoimmune Diseases / immunology
  • Autoimmune Diseases / therapy
  • Diabetes Mellitus / immunology
  • Diabetes Mellitus / therapy*
  • Forecasting
  • Gene Expression
  • Genetic Therapy / methods*
  • Genetic Therapy / trends
  • Humans
  • Insulin / genetics
  • Islets of Langerhans Transplantation
  • Models, Animal
  • Stem Cell Transplantation


  • Insulin