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类风湿关节炎的基因治疗:治疗基因的选择策略。

Gene therapy in rheumatoid arthritis: Strategies to select therapeutic genes.

机构信息

Department of Immunology, School of Medicine, Tehran University of Medical Sciences, Tehran, Iran.

Molecular Immunology Research Centre, Tehran University of Medical Sciences, Tehran, Iran.

出版信息

J Cell Physiol. 2019 Aug;234(10):16913-16924. doi: 10.1002/jcp.28392. Epub 2019 Feb 26.

Abstract

Significant advances have been achieved in recent years to ameliorate rheumatoid arthritis (RA) in animal models using gene therapy approaches rather than biological treatments. Although biological agents serve as antirheumatic drugs with suppressing proinflammatory cytokine activities, they are usually accompanied by systemic immune suppression resulting from continuous or high systemic dose injections of biological agents. Therefore, gene transfer approaches have opened an interesting perspective to deliver one or multiple genes in a target-specific or inducible manner for the sustained intra-articular expression of therapeutic products. Accordingly, many studies have focused on gene transferring methods in animal models by using one of the available approaches. In this study, the important strategies used to select effective genes for RA gene therapy have been outlined. Given the work done in this field, the future looks bright for gene therapy as a new method in the clinical treatment of autoimmune diseases such as RA, and by ongoing efforts in this field, we hope to achieve feasible, safe, and effective treatment methods.

摘要

近年来,通过基因治疗方法而非生物治疗,在改善类风湿关节炎(RA)动物模型方面取得了重大进展。虽然生物制剂作为抗风湿药物具有抑制促炎细胞因子活性的作用,但由于持续或高剂量全身注射生物制剂,它们通常会伴随全身免疫抑制。因此,基因转移方法为以靶向或诱导方式递送一种或多种基因以持续关节内表达治疗产品开辟了一个有趣的视角。因此,许多研究都集中在通过使用现有的方法之一在动物模型中进行基因转移方法上。在这项研究中,概述了选择 RA 基因治疗有效基因的重要策略。考虑到该领域的工作,基因治疗作为 RA 等自身免疫性疾病临床治疗的新方法前景光明,并且通过该领域的持续努力,我们希望实现可行、安全和有效的治疗方法。

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