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RNAi是由小双链RNAs触发的序列特异性转录后基因沉默,RNAi具有锁定任何引起疾病或疾病相关蛋白表达的能力,有巨大的治疗应用前景。但以治疗为目的的siRNA特异、有效的递送仍是RNAi广泛治疗应用的主要障碍。本文就目前siRNA体内递送取得的研究进展进行综述,以有助于RNAi技术安全、有效的临床应用。
RNAi is a sequence-specific post-transcriptional gene silencing triggered by small double-stranded RNAs. RNAi has the ability to lock out any protein that causes disease or disease-related protein expression, and has tremendous therapeutic prospect. However, the specific and effective delivery of siRNAs for therapeutic purposes remains a major obstacle to the widespread use of RNAi. This review summarizes the current advances in siRNA delivery in vivo to facilitate safe and effective clinical use of RNAi technology.