Prospects for gene therapy of insulin-dependent diabetes mellitus

S. Efrat*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review


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.

Original languageEnglish
Pages (from-to)1401-1409
Number of pages9
Issue number12
StatePublished - 1998


  • Beta-cell lines
  • Conditional transformation
  • Gene transfer
  • Glucose- regulated promoters
  • Immunomodulation
  • Insulin biosynthesis
  • Insulin secretion
  • Islet regeneration
  • Proinsulin processing
  • Transplantation


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