基因治疗骨关节炎的概念、机制及问题

Concept, mechanism and problem of gene therapy for osteoarthritis

背景:特定基因在特定的损伤部位的表达可增强细胞再生。目的:总结基因治疗的概念,对基因治疗的策略和基因治疗骨关节炎的机制进行综述,介绍基因治疗的研究方向。方法:以"Gene therapy、Cartilage、Cartilage repair、Osteoarthritis、Expression vectors、mi RNA"等关键词,广泛查阅Pub Med、Science Direct等外文数据库中关于基因治疗骨关节炎及其相关内容的文献,并进行分析和总结。结果与结论:基因治疗骨关节炎可以应用到骨关节炎发病的各个环节,并对相关物质代谢产生影响,通过病毒或非病毒载体,把目的基因递送到靶细胞。目前基因治疗的重点在加快细胞修复和减少炎症反应方面,同时基因载体和目的基因的多种选择提供了更好的个体化治疗方案。临床上基因治疗骨关节炎的具体机制尚不明确,存在基因突变的可能性,需深入研究验证安全性。

BACKGROUND: Specific gene expression at specific injury sites enhances cell regeneration. OBJECTIVE: To summarize the concept of gene therapy, to review the strategies of gene therapy and the mechanism of gene therapy for osteoarthritis, and to introduce the research direction of gene therapy. METHODS: Extensive consulted the literature on osteoarthritis gene therapy and related content in foreign databases such as Pub Med and Science Direct. The key words were ―Gene therapy, Cartilage, Cartilage repair, Osteoarthritis, Expression vectors, miRNA’’. Above data were analyzed and summarized. RESULTS AND CONCLUSION: Gene therapy for osteoarthritis can be applied to all aspects of the pathogenesis of osteoarthritis and affect the metabolism of related substances. The target gene is delivered to the target cells through viral or non-viral vectors. At present, the focus of gene therapy is to accelerate cell repair and reduce inflammatory response. At the same time, multiple choices of gene vectors and target genes provide better individualized treatment options. The specific mechanism of gene therapy for osteoarthritis in clinic is not clear, and there is the possibility of gene mutation, which needs to be further studied to verify the safety.