Review

Diabetologia

, Volume 41, Issue 12, pp 1401-1409

First online:

Prospects for gene therapy of insulin-dependent diabetes mellitus

  • S. EfratAffiliated withDepartment of Molecular Pharmacology, Albert Einstein College of Medicine, Bronx, New York, USA and Department of Human Genetics and Molecular Medicine, Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel

Summary

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. [Diabetologia (1998) 41: 1401–1409]

Keywords Beta-cell lines conditional transformation gene transfer glucose-regulated promoters immunomodulation insulin biosynthesis insulin secretion islet regeneration proinsulin processing transplantation