Mathisen P M, Tuohy V K
Department of Immunology, Lerner Research Institute, The Cleveland Clinic Foundation, Ohio 44195, USA.
J Clin Immunol. 2000 Sep;20(5):327-33. doi: 10.1023/a:1006625412367.
Gene therapy traditionally has been associated with "gene replacement." where exogenous recombinant DNA is introduced ex vivo into somatic cells that are then introduced back into the patient as a way to correct an inherited genetic defect. However, several novel gene therapy strategies for treating autoimmune diseases recently have emerged. Strategies involving the use of several types of DNA vaccines, the application of various viral vectors, and the use of diverse cellular vectors have shown promise in inhibiting autoimmune-mediated inflammation and repairing tissue damaged as a result of autoimmune attack. In the current review, we examine and discuss the development and proposed use of emerging gene therapy strategies for the treatment of autoimmune disease with specific emphasis on experimental autoimmune encephalomyelitis (EAE), an animal model widely used in multiple sclerosis (MS) research.
传统上,基因治疗一直与“基因替代”相关联,即将外源性重组DNA在体外导入体细胞,然后再将这些体细胞重新导入患者体内,以此来纠正遗传性基因缺陷。然而,最近出现了几种用于治疗自身免疫性疾病的新型基因治疗策略。涉及使用多种类型DNA疫苗、应用各种病毒载体以及使用不同细胞载体的策略,在抑制自身免疫介导的炎症和修复因自身免疫攻击而受损的组织方面已显示出前景。在当前的综述中,我们研究并讨论了用于治疗自身免疫性疾病的新兴基因治疗策略的发展和拟用情况,特别强调实验性自身免疫性脑脊髓炎(EAE),这是一种广泛用于多发性硬化症(MS)研究的动物模型。