| 引用本文: | 赵云春,张丽,郑彩虹.siRNA体内递送的研究现状[J].中国现代应用药学,2013,30(12):1366-1373. |
| ZHAO Yunchun,ZHANG Li,ZHENG Caihong.Current Status of siRNA Delivery[J].Chin J Mod Appl Pharm(中国现代应用药学),2013,30(12):1366-1373. |
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| 摘要: |
| RNAi是用来沉默特定基因的一个强有力的研究工具，并为基因治疗策略带来新希望。目前已经用于治疗肿瘤、乙型肝炎、老年性黄斑变性等疾病。RNAi的效应分子为小分子干扰RNA(siRNA)，然而裸siRNA自身具有电负性、分子量大、极性强、半衰期短，以及容易被内源酶降解和肾小球滤过等缺陷，使其临床应用受到极大的限制。如何通过对siRNA进行化学修饰和载体构建，包括运用病毒性载体和非病毒性载体，以提高siRNA的体内稳定性，成为当前的研究重点。本文对siRNA结构的化学修饰、siRNA的载体递送以及临床试验等研究现状予以综述。 |
| 关键词: RNAi siRNA 体内递送 载体 临床试验 |
| DOI： |
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| 基金项目:国家自然科学基金资助项目(81173000) |
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| Current Status of siRNA Delivery |
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ZHAO Yunchun1, ZHANG Li2, ZHENG Caihong3
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1.College of Pharmaceutical Science, Zhejiang University of Technology, Hangzhou 310014, China;2.School of Medicine, Zhejiang University, Hangzhou 310006, China;3.Women’s Hospital, School of Medicine, Zhejiang University, Hangzhou 310006, China
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| Abstract: |
| RNAi is a powerful research tool used to silence specific genes. It brings a new hope for gene therapy strategy. At present, it is used to treat a variety of diseases as tumour, hepatitis B, exudative age-related macular degeneration and so on. RNAi’s effector molecule is small interference RNA (siRNA). However, because of its negative charge, large molecule weight, strong polarity, short plasma half-life, readily degraded by serum endonucleases and efficiently removed by glomerular filtration, the clinical application of naked siRNA is greatly limited. Both the chemical modification of siRNA and carriers of siRNA including viral vectors and non-viral vectors can improve the stability of siRNA in vivo. Here, with the latest developments of the chemical modification of siRNA, carriers of siRNA delivery, clinical research of siRNA are summarized in this review. |
| Key words: RNAi siRNA in vivo delivery vector clinic trial |
