Prospects for gene therapy of insulin-dependent diabetes mellitus

Diabetologia. 1998 Dec;41(12):1401-9. doi: 10.1007/s001250051085.


The application of gene therapy to Type I (insulin-dependent) diabetes mellitus awaits improvements in gene transfer technologies and the development of better tools for accurate diagnosis of pre-diabetic people. Identification of the most promising candidate genes for gene transfer requires further elucidation of the molecular events involved in beta-cell autoimmune destruction, islet ontogeny and differentiation, and beta-cell function. This review outlines a number of possible targets for gene therapy in Type I diabetes, which could help prevent the autoimmune damage to islets, induce islet regeneration, and restore insulin production through engineering of self non-beta cells or beta-cell transplantation. It also evaluates their potential merits and drawbacks.

Publication types

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

MeSH terms

  • Autoimmune Diseases / prevention & control
  • Diabetes Mellitus, Type 1 / prevention & control
  • Diabetes Mellitus, Type 1 / therapy*
  • Gene Transfer Techniques
  • Genetic Engineering
  • Genetic Therapy*
  • Humans
  • Insulin / biosynthesis
  • Insulin / genetics
  • Islets of Langerhans / immunology
  • Islets of Langerhans / physiology
  • Regeneration


  • Insulin