Targeting autoimmune diabetes with gene therapy.
Targeting autoimmune diabetes with gene therapy.
复制标题
通过基因疗法治疗自身免疫性糖尿病。
作者：
N. Giannoukakis;W. Rudert;P. Robbins;M. Trucco
胰岛素依赖型或1型糖尿病的自身免疫性质靶向胰腺的β细胞进行破坏，并导致终身承诺胰岛素替代疗法。尽管近年来胰岛素的制剂和剂量的数量变得更加复杂和更有效，但单独的胰岛素治疗无法预防肾病、视网膜病或血管和心脏病，这些疾病仍然发生在大量患者中。已经尝试了不同的方法来消除外源性胰岛素施用的需要。从历史上看，这些包括胰腺和胰岛移植，后来与旨在阻止针对胰岛的破坏性过程的治疗相结合。尽管在所有这些领域都取得了重大进展，但每种方法都面临着敌对的免疫反应，这种反应往往以胰岛的丧失而告终。基因治疗为基础的方法增加了一个新的层面的努力，旨在具体阻断免疫攻击对胰岛的遗传风险的个人（自身免疫）或免疫反应对移植的同种异体胰岛（排斥反应）。这项新技术可能在1型糖尿病的治疗和治愈中发挥重要作用。
The autoimmune nature of insulin-dependent, or type 1, diabetes targets the beta-cells of the pancreas for destruction and results in a lifelong commitment to insulin replacement therapy. Although the number of formulations and dosing of insulin have become more sophisticated and more efficient in recent years, insulin therapy alone is unable to prevent nephropathy, retinopathy, or vascular and heart disease, which still occur in a large number of patients. Different approaches have been attempted to eliminate the requirement of exogenous insulin administration. Historically, these have included pancreatic and islet transplants, which were later combined with treatments intended to halt the destructive process directed against the islets. Despite significant advances made in all of these areas, each approach faces a hostile immunological response that frequently ends with the loss of the islets. Gene therapy-based approaches add a new dimension to the efforts aimed at specifically blocking the immunological attack against the islets in genetically at-risk individuals (autoimmunity) or the immunological response against transplanted allogeneic islets (rejection). This new technology may have an important role in the therapy and cure of type 1 diabetes.