School of Material Science and Chemical Engineering, Harbin University of Science and Technology, Harbin 150040, China.
School of Life Science and Technology, Harbin Institute of Technology, Harbin 150001, China.
Int J Mol Sci. 2023 Feb 8;24(4):3375. doi: 10.3390/ijms24043375.
Gene therapy has attracted much attention because of its unique mechanism of action, non-toxicity, and good tolerance, which can kill cancer cells without damaging healthy tissues. siRNA-based gene therapy can downregulate, enhance, or correct gene expression by introducing some nucleic acid into patient tissues. Routine treatment of hemophilia requires frequent intravenous injections of missing clotting protein. The high cost of combined therapy causes most patients to lack the best treatment resources. siRNA therapy has the potential of lasting treatment and even curing diseases. Compared with traditional surgery and chemotherapy, siRNA has fewer side effects and less damage to normal cells. The available therapies for degenerative diseases can only alleviate the symptoms of patients, while siRNA therapy drugs can upregulate gene expression, modify epigenetic changes, and stop the disease. In addition, siRNA also plays an important role in cardiovascular diseases, gastrointestinal diseases, and hepatitis B. However, free siRNA is easily degraded by nuclease and has a short half-life in the blood. Research has found that siRNA can be delivered to specific cells through appropriate vector selection and design to improve the therapeutic effect. The application of viral vectors is limited because of their high immunogenicity and low capacity, while non-viral vectors are widely used because of their low immunogenicity, low production cost, and high safety. This paper reviews the common non-viral vectors in recent years and introduces their advantages and disadvantages, as well as the latest application examples.
基因治疗因其独特的作用机制、无毒和良好的耐受性而备受关注，它可以杀死癌细胞而不损伤健康组织。基于 siRNA 的基因治疗可以通过将某些核酸引入患者组织中来下调、增强或纠正基因表达。血友病的常规治疗需要频繁地静脉注射缺失的凝血蛋白。联合治疗的高昂成本导致大多数患者缺乏最佳的治疗资源。siRNA 治疗具有持久治疗甚至治愈疾病的潜力。与传统手术和化疗相比，siRNA 的副作用更少，对正常细胞的损伤更小。退行性疾病的现有治疗方法只能缓解患者的症状，而 siRNA 治疗药物可以上调基因表达、修饰表观遗传变化并阻止疾病进展。此外，siRNA 在心血管疾病、胃肠道疾病和乙型肝炎中也发挥着重要作用。然而，游离的 siRNA 很容易被核酸酶降解，并且在血液中的半衰期很短。研究发现，通过适当的载体选择和设计，siRNA 可以被递送到特定的细胞中，从而提高治疗效果。病毒载体的应用受到其高免疫原性和低容量的限制，而非病毒载体则因其低免疫原性、低成本和高安全性而被广泛应用。本文综述了近年来常见的非病毒载体，并介绍了它们的优缺点以及最新的应用实例。
