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自身免疫性疾病的基因治疗。

Gene therapy for autoimmune diseases.

作者信息

Furlan Roberto, Butti Erica, Pluchino Stefano, Martino Gianvito

机构信息

Neuroimmunology Unit, DIBIT, Department of Neurology and Neurophysiology, San Raffaele Scientific Institute, Via Olgettina 58, 20132 Milan, Italy.

出版信息

Curr Opin Mol Ther. 2004 Oct;6(5):525-36.

Abstract

Autoimmune diseases are threatening an increasing number of patients in developed countries, representing one of the major causes of disability and an enormous social cost. Current therapies mainly treat the symptoms of autoimmune diseases and are only partially able to interfere with disease evolution, and therefore decrease the degree of physical impairment. Thus, the development of new therapeutic strategies is imperative. This review focuses on gene therapy, as one possible alternative approach to the treatment of autoimmune disorders. The potential of gene therapy to specifically target tissues affected by autoimmune aggression, and its ability to interfere with the destructive pathogenic process while providing functional replacement and fostering reparative mechanisms will be emphasized. Gene therapy studies in experimental models of diabetes, rheumatoid arthritis and multiple sclerosis are reviewed.

摘要

自身免疫性疾病正在威胁发达国家越来越多的患者,是导致残疾的主要原因之一,并带来巨大的社会成本。目前的疗法主要治疗自身免疫性疾病的症状,只能部分干扰疾病的发展,因此只能降低身体损伤的程度。因此,开发新的治疗策略势在必行。本综述重点关注基因治疗,这是一种治疗自身免疫性疾病的可能替代方法。将强调基因治疗特异性靶向受自身免疫攻击影响的组织的潜力,以及其在提供功能替代和促进修复机制的同时干扰破坏性致病过程的能力。本文综述了糖尿病、类风湿性关节炎和多发性硬化症实验模型中的基因治疗研究。

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