《生命科学》 2014, 26(4): 392-399

介导siRNA传递的非病毒载体及其研究进展

李泽豪¹，任小元¹，王世兵¹，孔祥东^1，2*

(1 浙江理工大学生命科学学院生物材料研究所，杭州 310018；2 浙江理工大学材料与
    纺织学院，教育部先进纺织材料和制备技术重点实验室，杭州 310018)

摘　要：RNA 干扰(RNAi) 是一种疾病治疗的新途径，其中小干扰RNA (siRNA) 可破坏mRNA 的完整性，抑制疾病相关基因的表达，而开发安全有效的siRNA 传递载体仍面临巨大挑战。相较于病毒载体系统，非病毒载体系统安全性高、负载量大、生物相容性好，更适用于疾病治疗。综述了可用于siRNA 传递的一些非病毒载体( 包括脂质体、细胞穿膜肽、水凝胶、树枝状大分子、磷酸钙、碳纳米管等)，阐述了载体与siRNA 的结合，靶向疾病组织或细胞并诱导特异基因沉默的机制、瓶颈问题等。

Research advances in non-viral-based siRNA delivery systems

LI Ze-Hao¹， REN Xiao-Yuan¹， WANG Shi-Bing¹， KONG Xiang-Dong^1，2*

(1 Institute of Biomaterials， College of Life Sciences， Zhejiang Sci-Tech University， Hangzhou 310018， China; 2 Key Laboratory of Advanced Textile Materials and Manufacturing Technology of Ministry of Education，College of Materials and Textile， Zhejiang Sci-Tech University， Hangzhou 310018， China)

Abstract: RNA interference (RNAi) is a new mechanism triggered by small interfering RNA (siRNA)， which represents a promising gene therapy strategy by destroying the integrity of the complementary mRNA in the cytoplasm， causing sequence-specific posttranscriptional gene silencing. Systemic siRNA therapy is hampered by inefficient delivery of its carrier. Compared with viral-based delivery systems， non-viral-based delivery systems are safer， and have higher load， greater cellular compatibility， so they are more suitable for clinical application. In general， the non-viral carriers include cation lipid-based carriers， cation cell-penetrating peptides， dendrimers， cation polymers， inorganic nanomaterials. This review describes the recent types and advantage in siRNA delivery using non-viral-based siRNA delivery systems.