This work was supported by a grant from The National Natural Science Foundation of China(30500608, 30973481)
小分子干扰RNA(small interfering RNA,siRNA)因能快速抑制哺乳动物特定基因的表达而用于各种疾病的治疗,然而选择合适的载体将siRNA安全有效地转运进入靶细胞仍是制约siRNA应用于临床治疗的重要因素.越来越多的转运载体被开发出来,其中包括针对细胞表面蛋白的适配子(aptamer).Aptamer是一种能与靶分子高特异性和高亲和结合的寡核苷酸,已经越来越多地用于疾病的诊断和治疗.Aptamer作为载体介导siRNA转运可提高治疗的靶向性并减少副作用,这将为siRNA应用于临床靶向治疗提供一种特异有效的途径.目前,已经发现几种aptamers可以介导siRNA的转运,如anti-PSMA aptamer,anti-HIV gp120 aptamer,anti-CD4 aptamer等.本文将综述aptamer介导siRNA转运的最新研究进展.
The ability of small interfering RNA (siRNA) to inhibit mammalian gene expression is being exploited as a new class of therapeutics for a variety of diseases.However, the efficient and safe delivery of siRNAs into specific cell populations is still the principal challenge in the clinical development of RNAi therapeutics.Many potential delivery vehicles and vectors have been explored including the aptamers targeting cell surface proteins.Selected nucleic acid binding species (aptamers) are high affinity and specificity for their targets, and they have been effectively applied in targeted therapy and diagnostics of diseases.The aptamer-based delivery of siRNAs can often enhance the therapeutic efficacy and reduce the unwanted off-target effects of siRNAs.Nowadays, some kinds of aptamers are able to mediate the delivery of siRNA, such as anti-PSMA aptamer, anti-gp120 aptamer. I will review the latest progress about aptamer mediated siRNA.
谭燕,张兴梅.综述: 适配子介导的siRNA转运[J].生物化学与生物物理进展,2012,39(5):410-415
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