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Updated: Feb 10, 2026

Isolation of Small Noncoding RNAs from Human Serum
Published on: June 19, 2014
Therapeutic Potential of Small Activating RNAs (saRNAs) in Human Cancers
Sorah Yoon1, John J Rossi1,2
1Department of Molecular and Cellular Biology, Beckman Research Institute of City of Hope, Duarte, California, United States.
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.
Insights
Small activating RNAs (saRNAs) are emerging as powerful gene regulators for oligotherapeutics. This review details saRNA mechanisms, therapeutic progress in cancer, and aptamer-based delivery advancements.
Area of Science:
- Molecular Biology
- RNA Therapeutics
- Gene Regulation
Background:
- RNA molecules are recognized for their potent gene expression control capabilities.
- Advanced RNA-based regulators are under development as novel oligotherapeutics.
- Small activating RNAs (saRNAs) show significant promise for treating human diseases.
Purpose of the Study:
- To review the intracellular mechanism of saRNA action.
- To discuss the therapeutic advancements of saRNAs in preclinical and clinical settings.
- To explore the latest developments in targeted delivery of saRNAs using aptamers.
Main Methods:
- Review of current literature on saRNA mechanisms and therapeutic applications.
- Analysis of preclinical and clinical trial data for saRNA-based cancer therapies.
- Examination of aptamer technology for targeted delivery of RNA therapeutics.
Main Results:
- Detailed explanation of the intracellular working model of saRNAs.
- Overview of numerous saRNAs developed for various cancers in preclinical studies.
- Highlight of a specific saRNA targeting C/EBPα currently in clinical trials for liver cancer.
Conclusions:
- saRNAs represent a promising class of therapeutic agents.
- Continued research into saRNA mechanisms and delivery is crucial for clinical success.
- Aptamer-mediated delivery offers a promising strategy for enhancing saRNA therapeutic efficacy.
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