Related Experiment Video
Updated: May 10, 2026
09:06
MicroRNA Amplification and Recognition through Locked-nucleic-acid In situ Hybridization as a Novel Detection and Quantification Method
Published on: October 7, 2025
Small molecules modulating biogenesis or processing of microRNAs with therapeutic potentials
1School of Chemistry and Chemical Engineering, State Key Lab of Analytical Chemistry for Life Science, Nanjing University, China.
Current Medicinal Chemistry
|June 11, 2013
Summary
Small molecules can regulate microRNAs (miRNAs), which are key gene regulators. This review covers small-molecule miRNA modulators, their screening, mechanisms, and therapeutic potential for diseases.
Area of Science:
- Molecular Biology
- Genetics
- Pharmacology
Background:
- MicroRNAs (miRNAs) are crucial regulators of gene expression at the post-transcriptional level.
- miRNAs influence nearly all genetic pathways and are vital in physiological and pathological processes.
- Dysregulation of miRNAs is implicated in various diseases, highlighting their therapeutic relevance.
Purpose of the Study:
- To review small molecules that modulate microRNA (miRNA) biogenesis or function.
- To emphasize screening methods for identifying these small-molecule regulators.
- To discuss proposed mechanisms of action and therapeutic potentials of identified compounds.
Main Methods:
- Literature review of studies reporting small-molecule regulators of miRNAs.
- Analysis of screening systems used for identifying miRNA modulators.
- Evaluation of proposed mechanisms and demonstrated therapeutic applications.
Main Results:
- Several small-molecule regulators of miRNAs have been identified using recent screening systems.
- These molecules act as specific or universal regulators of miRNA activity.
- Therapeutic potential of these small molecules in disease contexts has been demonstrated.
Conclusions:
- Small molecules offer promising tools for probing miRNA-mediated networks.
- Disease-related miRNA modulators hold significant potential as novel therapeutic agents.
- Further research into small-molecule miRNA regulators could lead to new treatment strategies.
Related Concept Videos
01:22MicroRNAs
MicroRNA (miRNA) are short, regulatory RNA transcribed from introns (non-coding regions of a gene) or intergenic regions (stretches of DNA present between genes). Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself, forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA...
01:22MicroRNAs
MicroRNA (miRNA) are short, regulatory RNA transcribed from introns—non-coding regions of a gene—or intergenic regions—stretches of DNA present between genes. Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA ends...
01:22MicroRNAs
MicroRNA (miRNA) are short, regulatory RNA transcribed from introns—non-coding regions of a gene—or intergenic regions—stretches of DNA present between genes. Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA ends...
02:30siRNA - Small Interfering RNAs
Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
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 ATP-dependent...
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 ATP-dependent...
01:23RNA Interference
RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
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...
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...
01:23RNA Interference
RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
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...
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...