Chistiakov Dimitry A, Tyurina Inna
a Assistant Professor, University of Pittsburgh Medical Center, Department of Pathology, 3550 Terrace Street, Pittsburgh, PA, 15261, USA.
b Executive Manager and Consultant, Public Relations and Consulting Group 'Imya', 8th Tekstilschikov Street 11, 109129, Moscow, Russia.
Expert Rev Endocrinol Metab. 2007 Jan;2(1):27-34. doi: 10.1586/17446651.2.1.27.
Insulin gene therapy is an approach that might overcome the weakness of islet cell therapy owing to its vulnerability to autoimmune attack. There are several mandatory conditions for successful insulin gene therapy. Efficient insulin gene therapy should have an effective insulin gene delivery mechanism, a system of regulation of the insulin biosynthesis that responds to glucose within extremely narrow physiological limits, a system of insulin processing into its active form and a choice of appropriate target cells, which possess biochemical characteristics similar to β cells, but are not targets for β-cell-specific self-reactivity. In this article, advantages and disadvantages of non-β-cell types that are most likely to be used for generating surrogate insulin-producing β cells are compared. Current achievements in insulin gene therapy are critically evaluated and future challenges are discussed.
胰岛素基因疗法是一种可能克服胰岛细胞疗法弱点的方法，因为胰岛细胞疗法易受自身免疫攻击。成功的胰岛素基因疗法有几个必要条件。高效的胰岛素基因疗法应具备有效的胰岛素基因递送机制、在极其狭窄的生理范围内对葡萄糖作出反应的胰岛素生物合成调节系统、将胰岛素加工成活性形式的系统以及选择具有与β细胞相似生化特性但不是β细胞特异性自身反应靶点的合适靶细胞。在本文中，比较了最有可能用于生成替代胰岛素分泌β细胞的非β细胞类型的优缺点。对胰岛素基因疗法的当前成果进行了批判性评估，并讨论了未来的挑战。
