Efrat S
Department of Molecular Pharmacology, Albert Einstein College of Medicine, Bronx, New York, USA.
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.
将基因疗法应用于I型（胰岛素依赖型）糖尿病，尚需改进基因转移技术，并开发出更优工具以准确诊断糖尿病前期患者。要确定最具潜力的基因转移候选基因，需要进一步阐明参与β细胞自身免疫性破坏、胰岛个体发生与分化以及β细胞功能的分子事件。本综述概述了I型糖尿病基因治疗的一些可能靶点，这些靶点有助于预防胰岛的自身免疫损伤、诱导胰岛再生，并通过改造自身非β细胞或进行β细胞移植来恢复胰岛素分泌。同时还评估了它们潜在的优缺点。
