title: Prospects for gene therapy of insulin-dependent diabetes mellitus.
abstract: 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.
authors.affiliation: Department of Molecular Pharmacology, Albert Einstein College of Medicine, Bronx, New York, USA.
authors.forename: S
authors.initials: S
authors.lastname: Efrat
mesh_terms: D001327:Autoimmune Diseases / Q000517:prevention & control; D003922:Diabetes Mellitus, Type 1 / Q000517:prevention & control / Q000628:therapy*; D018014:Gene Transfer Techniques; D005818:Genetic Engineering; D015316:Genetic Therapy; D006801:Humans; D007328:Insulin / Q000096:biosynthesis / Q000235:genetics; D007515:Islets of Langerhans / Q000276:immunology / Q000502:physiology; D012038:Regeneration
chemical_list: D007328:Insulin