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Updated: May 12, 2026

MicroRNA Amplification and Recognition through Locked-nucleic-acid In situ Hybridization as a Novel Detection and Quantification Method
Published on: October 7, 2025
RNAi therapeutics and applications of microRNAs in cancer treatment
Keita Uchino1, Takahiro Ochiya, Fumitaka Takeshita
1Division of Molecular and Cellular Medicine, National Cancer Center Research Institute, 1-1, Tsukiji 5-chome, Chuo-ku, Tokyo 104-0045, Japan.
Abstract:
RNA interference-based therapies are proving to be powerful tools for combating various diseases, including cancer. Scientists are researching the development of safe and efficient systems for the delivery of small RNA molecules, which are extremely fragile in serum, to target organs and cells in the human body. A dozen pre-clinical and clinical trials have been under way over the past few years involving biodegradable nanoparticles, lipids, chemical modification and conjugation. On the other hand, microRNAs, which control the balance of cellular biological processes, have been studied as attractive therapeutic targets in cancer treatment. In this review, we provide an overview of RNA interference-based therapeutics in clinical trials and discuss the latest technology for the systemic delivery of nucleic acid drugs. Furthermore, we focus on dysregulated microRNAs in human cancer, which have progressed in pre-clinical trials as therapeutic targets, and describe a wide range of strategies to control the expression levels of endogenous microRNAs. Further development of RNA interference technologies and progression of clinical trials will contribute to the achievement of practical applications of nucleic acid drugs.
Insights
RNA interference therapies show promise for cancer treatment. Researchers are developing advanced delivery systems for fragile RNA molecules and targeting microRNAs to advance nucleic acid drug applications.
Area of Science:
- Biomedical Science
- Molecular Biology
- Oncology
Background:
- RNA interference (RNAi) therapies are emerging as potent treatments for diseases, notably cancer.
- Developing safe and efficient delivery systems for fragile small RNA molecules to target cells is a key challenge.
- MicroRNAs (miRNAs), crucial regulators of cellular processes, are recognized as significant therapeutic targets in oncology.
Purpose of the Study:
- To review RNAi-based therapeutics currently in clinical trials.
- To discuss cutting-edge technologies for systemic delivery of nucleic acid drugs.
- To explore the role of dysregulated microRNAs in human cancers and strategies for their therapeutic modulation.
Main Methods:
- Overview of RNA interference-based therapeutics in clinical trials.
- Discussion of systemic delivery technologies for nucleic acid drugs.
- Focus on microRNA dysregulation in cancer and methods for controlling miRNA expression.
Main Results:
- A dozen pre-clinical and clinical trials are investigating RNAi therapies using various delivery methods like nanoparticles and chemical modifications.
- MicroRNAs have shown promise as therapeutic targets in pre-clinical cancer studies.
- Diverse strategies are being developed to modulate endogenous microRNA levels.
Conclusions:
- Advancements in RNA interference technologies and clinical trials are paving the way for practical applications of nucleic acid drugs.
- Effective delivery systems are crucial for the success of RNAi-based therapies.
- Targeting microRNAs offers a promising avenue for novel cancer treatments.
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This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
RNA Interference
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...

