Department of Immunology, Monash Central Clinical School, Monash University, Victoria, Australia.
Mol Pharm. 2011 Oct 3;8(5):1488-94. doi: 10.1021/mp2001523. Epub 2011 Jul 13.
A key function of the immune system is to protect us from foreign pathogens such as viruses, bacteria, fungi and multicellular parasites. However, it is also important in many other aspects of human health such as cancer surveillance, tissue transplantation, allergy and autoimmune disease. Autoimmunity can be defined as a chronic immune response that targets self-antigens leading to tissue pathology and clinical disease. Autoimmune diseases, as a group of diseases that include type 1 diabetes, multiple sclerosis, rheumatoid arthritis and systemic lupus erythematosus, have no effective cures, and treatment is often based on long-term broad-spectrum immunosuppressive regimes. While a number of strategies aimed at providing disease specific treatments are being explored, one avenue of study involves the use of hematopoietic stem cells to promote tolerance. In this manuscript, we will review the literature in this area but in particular examine the relatively new experimental field of gene therapy and hematopoietic stem cell transplantation as a molecular therapeutic strategy to combat autoimmune disease.
免疫系统的一个主要功能是保护我们免受外来病原体的侵害，如病毒、细菌、真菌和多细胞寄生虫。然而，它在人类健康的许多其他方面也很重要，如癌症监测、组织移植、过敏和自身免疫性疾病。自身免疫可以被定义为一种针对自身抗原的慢性免疫反应，导致组织病理学和临床疾病。作为一组疾病，包括 1 型糖尿病、多发性硬化症、类风湿性关节炎和系统性红斑狼疮，自身免疫性疾病没有有效的治疗方法，治疗通常基于长期的广谱免疫抑制治疗方案。虽然正在探索许多旨在提供针对疾病的治疗策略的方法，但一个研究途径涉及使用造血干细胞来促进耐受。在本文中，我们将回顾这一领域的文献，但特别要研究相对较新的基因治疗和造血干细胞移植的实验领域，作为一种对抗自身免疫性疾病的分子治疗策略。
