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。
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.
出处
《生命科学》
CSCD
2014年第4期392-399,共8页
Chinese Bulletin of Life Sciences
基金
浙江理工大学521人才培养计划
浙江省自然科学基金项目(Y207217)
国家自然科学基金项目(51272236
51002139
51172207)
