| 中文关键词: 小激活RNAs 基因激活 恶性肿瘤 |
| 英文关键词:small activating RNAs gene activation malignant neoplasms |
| 基金项目:浙江省自然科学基金（LY15H160035） |
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| 中文摘要: |
| 摘 要：小激活RNAs（saRNA）是一类非编码RNA，具有与小干扰RNA（siRNA）相同的结构和化学组分。尽管它们具有完全相反的生物学功能，但是saRNA能介导细胞中相应靶基因的转录激活。最近的一系列研究为RNAa提供了明确的证据，而开展的第一个RNAa Ⅰ期临床试验表明，运用特殊介质传递saRNA药物能实现对患者疾病的稳定或肿瘤消退，并具有良好的耐受性。因此，借助于saRNAs特殊的作用机制和特征，RNAa可能成为一些疾病的全新治疗方法。 |
| 英文摘要: |
| Abstract：In recent years，small RNA-mediated gene regulation in the treatment of various diseases has attracted widespread attention. Small activating RNAs(saRNAs) are non-coding RNAs that share the same structural and chemical components as small interfering RNAs(siRNAs). Although they have different biological functions，saRNAs can activate the transcriptional expression of sequence-specific mRNA of the corresponding target gene in cells. Recent studies have provided clear evidence for RNAa，and the first phaseⅠclinical trials of RNAa have shown that the use of specific mediators to deliver saRNA drugs can achieve disease stable or tumor regression，and the patients are well tolerated. Therefore，with the special mechanism and characteristics of saRNAs，saRNAs may become a new approach of treatment for some diseases. This article reviews the recent advances in the basic and clinical research of saRNAs in tumors. |
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