title: Preventing type 1 diabetes mellitus: the promise of gene therapy.
abstract: Type 1 (insulin-dependent) diabetes mellitus is an autoimmune disease that has no cure. Closed-loop insulin administration strategies and approaches for replacement of the insulin-producing beta cells may offer improved treatments, which could delay or prevent diabetes complications. In the long run, however, prevention of type 1 diabetes in susceptible individuals represents the best chance for reducing the toll of the disease. Prevention of type 1 diabetes will require reliable methods for early diagnosis of predisposition to the disease, using improved genetic and serological screening on a wide scale. Identification of the primary antigenic target(s) for autoimmunity will allow intervention in prediabetes stages aimed at the induction of antigen-specific tolerance. In addition to manipulation of the immune system, the susceptibility of beta cells to autoimmunity could be reduced. A number of genes have been shown to increase beta-cell resistance to immune effector molecules in animal models and cultured beta-cell lines. These genes could be used for preventive gene therapy of type 1 diabetes mellitus if expressed in beta cells prior to the onset of autoimmune destruction. This prospect depends on the development of safe and efficient vectors, and approaches for cell-specific targeting of these vectors to beta cells in vivo.
authors.affiliation: Department of Human Genetics and Molecular Medicine, Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel. sefrat@post.tau.ac.il
authors.forename: Shimon
authors.initials: S
authors.lastname: Efrat
mesh_terms: D000818:Animals; D003922:Diabetes Mellitus, Type 1 / Q000235:genetics* / Q000517:prevention & control* / Q000628:therapy; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D006801:Humans