Related Experiment Video
Updated: Nov 10, 2025

MicroRNA Amplification and Recognition through Locked-nucleic-acid In situ Hybridization as A Novel Detection and Quantification Method
Published on: October 7, 2025
microRNAs as Novel Therapeutics in Cancer
Giulia Romano1, Mario Acunzo1, Patrick Nana-Sinkam1
1Department of Internal Medicine, Division of Pulmonary Diseases and Critical Care Medicine, Virginia Commonwealth University, Richmond, VA 23298, USA.
Abstract:
In the last 20 years, the functional roles for miRNAs in gene regulation have been well established. MiRNAs act as regulators in virtually all biological pathways and thus have been implicated in numerous diseases, including cancer. They are particularly relevant in regulating the basic hallmarks of cancer, including apoptosis, proliferation, migration, and invasion. Despite the substantial progress made in identifying the molecular mechanisms driving the deregulation of miRNAs in cancer, the clinical translation of these important molecules to therapy remains in its infancy. The paucity of vehicles available for the safe and efficient delivery of miRNAs and ongoing concerns for toxicity remain major obstacles to clinical application. Novel formulations and the development of new vectors have significantly improved the stability of oligonucleotides, increasing the effectiveness of therapy. Furthermore, the use of specific moieties for delivery in target tissues or cells has increased the specificity of treatment. The use of new technologies has allowed small but important steps toward more specific therapeutic delivery in tumor tissues and cells. Although a long road remains, the path ahead holds great potential. Currently, a few miRNA drugs are under investigation in human clinical trials with promising results ahead.
Insights
MicroRNAs (miRNAs) are key gene regulators in cancer, but their clinical use is limited by delivery challenges. Advances in novel formulations and targeted delivery vectors are paving the way for effective miRNA cancer therapies.
Area of Science:
- Molecular Biology
- Genetics
- Oncology
Background:
- MicroRNAs (miRNAs) are established regulators of gene expression across biological pathways.
- miRNAs play critical roles in cancer hallmarks like apoptosis, proliferation, migration, and invasion.
- Deregulation of miRNAs is implicated in numerous cancers, highlighting their therapeutic potential.
Purpose of the Study:
- To review the progress and challenges in translating miRNA research into clinical cancer therapy.
- To discuss advancements in miRNA delivery vehicles and targeted therapeutic strategies.
- To highlight the potential of miRNA-based drugs in cancer treatment.
Main Methods:
- Review of recent literature on miRNA function in cancer.
- Analysis of novel formulations and vectors for oligonucleotide delivery.
- Examination of targeted delivery strategies for miRNA therapeutics.
- Overview of current clinical trials involving miRNA drugs.
Main Results:
- Significant progress has been made in understanding miRNA roles in cancer.
- Novel formulations and vectors have improved miRNA stability and therapeutic effectiveness.
- Targeted delivery moieties enhance specificity in tumor cells.
- Early clinical trials show promising results for miRNA-based cancer drugs.
Conclusions:
- Despite challenges in safe and efficient delivery, miRNA-based cancer therapies show great potential.
- Advancements in delivery technologies are overcoming major obstacles to clinical application.
- Several miRNA drugs are progressing through clinical trials, indicating a promising future for this therapeutic modality.
Related Concept Videos
MicroRNAs
MicroRNAs
siRNA - Small Interfering RNAs
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...
Targeted Cancer Therapies
There are several types of targeted therapies against...
lncRNA - Long Non-coding RNAs
Microorganisms in Medicine and Therapeutics

