小分子激活RNA（saRNAs）在人类癌症中的治疗潜力
Therapeutic Potential of Small Activating RNAs (saRNAs) in Human Cancers.
作者信息
Yoon Sorah, Rossi John J
机构信息
Department of Molecular and Cellular Biology, Beckman Research Institute of City of Hope, Duarte, California, United States.
Irell and Manella Graduate School of Biological Sciences, Beckman Research Institute of City of Hope, Duarte, California, United States.
出版信息
Curr Pharm Biotechnol. 2018;19(8):604-610. doi: 10.2174/1389201019666180528084059.
BACKGROUND
RNA is increasingly recognized as a powerful molecule that can be used to control gene expression. Sophisticated, well-engineered RNA-based regulators are being developed as oligotherapeutics.
METHODS
In particular, small activating RNAs (saRNAs) are promising therapeutic options for targeting human diseases. Numerous saRNAs targeting multiple cancers have been developed in preclinical models. One saRNA targeting C/EBPα is currently undergoing clinical trials in liver cancer.
RESULTS AND CONCLUSION
In this review, we describe the current working model of the intracellular mechanism of saRNA, discuss the recent progress of saRNA therapeutics in preclinical and clinical trials, and current advances in targeted delivery using aptamers in detail.
背景
RNA越来越被认为是一种可用于控制基因表达的强大分子。复杂的、精心设计的基于RNA的调节剂正在被开发为寡核苷酸疗法。
方法
特别是，小激活RNA（saRNAs）是针对人类疾病的有前景的治疗选择。在临床前模型中已经开发了许多针对多种癌症的saRNAs。一种靶向C/EBPα的saRNA目前正在肝癌中进行临床试验。
结果与结论
在本综述中，我们描述了saRNA细胞内机制的当前工作模型，详细讨论了saRNA疗法在临床前和临床试验中的最新进展，以及使用适体进行靶向递送的当前进展。
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