小干扰RNA
RNA干扰
基因沉默
反式siRNA
计算生物学
生物
生物信息学
核糖核酸
纳米技术
基因
遗传学
材料科学
作者
Can Sarısözen,Giuseppina Salzano,Vladimir P. Torchilin
标识
DOI:10.1515/bmc-2015-0019
摘要
In the 1990s an unexpected gene-silencing phenomena in plants, the later called RNA interference (RNAi), perplexed scientists. Following the proof of activity in mammalian cells, small interfering RNAs (siRNAs) have quickly crept into biomedical research as a new powerful tool for the potential treatment of different human diseases based on altered gene expression. In the past decades, several promising data from ongoing clinical trials have been reported. However, despite surprising successes in many pre-clinical studies, concrete obstacles still need to be overcome to translate therapeutic siRNAs into clinical reality. Here, we provide an update on the recent advances of RNAi-based therapeutics and highlight novel synthetic platforms for the intracellular delivery of siRNAs.
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