Sindhu Rakesh K, Madaan Piyush, Chandel Parteek, Akter Rokeya, Adilakshmi Griddaluri, Rahman Md Habibur
Chitkara College of Pharmacy, Chitkara University, Punjab, India.
Department of Pharmacy, Jagannath University, Sadarghat, Dhaka-1100, Bangladesh.
Curr Gene Ther. 2022;22(3):245-261. doi: 10.2174/1566523221666210916113609.
Autoimmune diseases are the diseases that result due to the overactive immune response, and comprehend systemic autoimmune diseases like Rheumatoid Arthritis (RA), Sjӧgren's Syndrome (SS), and organ-specific autoimmune diseases like type-1 diabetes mellitus (T1DM), Myasthenia Gravis (MG), and Inflammatory Bowel Disease (IBD). Currently, there is no long-term cure; but, several treatments exist which retard the evolution of the disease, embracing gene therapy, which has been scrutinized to hold immense aptitude for the management of autoimmune diseases.
The review highlights the pathogenic mechanisms and genes liable for the development of autoimmune diseases, namely T1DM, type-2 diabetes mellitus (T2DM), RA, SS, IBD, and MG. Furthermore, the review focuses on investigating the outcomes of delivering the corrective genes with their specific viral vectors in various animal models experiencing these diseases to determine the effectiveness of gene therapy.
Numerous review and research articles emphasizing the tremendous potential of gene therapy in the management of autoimmune diseases were procured from PubMed, MEDLINE, Frontier, and other databases and thoroughly studied for writing this review article.
The various animal models that experienced treatment with gene therapy have displayed regulation in the levels of proinflammatory cytokines, infiltration of lymphocytes, manifestations associated with autoimmune diseases, and maintained equilibrium in the immune response, thereby compete with the progression of autoimmune diseases.
Gene therapy has revealed prodigious aptitude in the management of autoimmune diseases in various animal studies, but further investigation is essential to combat the limitations associated with it and before employing it on humans.
自身免疫性疾病是由于免疫反应过度活跃而导致的疾病，包括类风湿性关节炎（RA）、干燥综合征（SS）等全身性自身免疫性疾病，以及1型糖尿病（T1DM）、重症肌无力（MG）和炎症性肠病（IBD）等器官特异性自身免疫性疾病。目前尚无长期治愈方法；但是，存在几种延缓疾病进展的治疗方法，包括基因治疗，该方法已被仔细研究，在自身免疫性疾病的治疗中具有巨大潜力。
本综述重点介绍导致自身免疫性疾病（即T1DM、2型糖尿病（T2DM）、RA、SS、IBD和MG）发生的致病机制和相关基因。此外，本综述着重研究在患有这些疾病的各种动物模型中，使用其特定病毒载体递送校正基因的效果，以确定基因治疗的有效性。
从PubMed、MEDLINE、Frontier和其他数据库中获取了大量强调基因治疗在自身免疫性疾病治疗中巨大潜力的综述和研究文章，并对其进行了深入研究，以撰写本综述文章。
接受基因治疗的各种动物模型在促炎细胞因子水平调节、淋巴细胞浸润、自身免疫性疾病相关表现以及免疫反应维持平衡方面均有体现，从而对抗自身免疫性疾病的进展。
在各种动物研究中，基因治疗在自身免疫性疾病的治疗中已显示出巨大潜力，但在克服与之相关的局限性并应用于人类之前，还需要进一步研究。
