Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Experimental RNAi02:15

Experimental RNAi

6.2K
RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
6.2K
siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

17.0K
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...
17.0K
RNA Interference01:23

RNA Interference

26.4K
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...
26.4K
Types of RNA01:23

Types of RNA

64.8K
Overview
Three main types of RNA are involved in protein synthesis: messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). These RNAs perform diverse functions and can be broadly classified as protein-coding or non-coding RNA. Non-coding RNAs play important roles in the regulation of gene expression in response to developmental and environmental changes. Non-coding RNAs in prokaryotes can be manipulated to develop more effective antibacterial drugs for human or animal use.
RNA...
64.8K
Leaky Scanning02:28

Leaky Scanning

5.2K
During most eukaryotic translation processes, the small 40S ribosome subunit scans an mRNA from its 5' end until it encounters the first start AUG codon. The large 60S ribosomal subunit then joins the smaller one to initiate protein synthesis. The location of the translation initiation is largely determined by the nucleotides near the start codon as there may be multiple translation initiation sites present on the mRNA.  Marilyn Kozak discovered that the sequence RCCAUGG (where R...
5.2K
Ribozymes02:47

Ribozymes

12.5K
The term ribozyme is used for RNA that can act as an enzyme. Ribozymes are mainly found in selected viruses, bacteria, plant organelles, and lower eukaryotes. Ribozymes were first discovered in 1982 when Tom Cech’s laboratory observed Group I introns acting as enzymes. This was shortly followed by the discovery of another ribozyme, Ribonulcease P, by Sid Altman’s laboratory. Both Cech and Altman received the Nobel Prize in chemistry in 1989 for their work on ribozymes.
Ribozymes can...
12.5K

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Conserved 3' stem-loop structures enable comprehensive analysis of bacterial transcription termination in metagenomes.

Microbiome·2026
Same author

Towards the construction of a virtual yeast.

Nature·2026
Same author

A Biomimetic Single-Atom Nanozyme With a Substrate Pocket for Accurate and Continuous Sweat Glucose Monitoring.

Advanced materials (Deerfield Beach, Fla.)·2026
Same author

Elevated Visceral Adiposity Index is Associated with Reproductive Endocrine Characteristics, and Adverse Pregnancy Outcomes in Chinese Women with Polycystic Ovary Syndrome.

International journal of women's health·2026
Same author

Tryptamine from wake-active monoaminergic neurons regulates sleep homeostasis.

Nature neuroscience·2026
Same author

Infiltrating monocyte-derived macrophages does not survive long term in stroke brain despite their dominance in the acute ischemic core and myeloid derived IGF-1 play dichotomous roles in stroke recovery.

Genome medicine·2026

Related Experiment Video

Updated: Sep 10, 2025

Using In Vitro and In-cell SHAPE to Investigate Small Molecule Induced Pre-mRNA Structural Changes
11:58

Using In Vitro and In-cell SHAPE to Investigate Small Molecule Induced Pre-mRNA Structural Changes

Published on: January 30, 2019

8.4K

Discovery of RNA-Targeting Small Molecules: Challenges and Future Directions.

Zhengguo Cai1, Hongli Ma2, Fengcan Ye3

  • 1DP Technology Beijing China.

Medcomm
|August 27, 2025
PubMed
Summary

RNA-targeting small molecules offer new hope for difficult-to-treat diseases. Advances in structure determination, computational methods, and AI are accelerating the discovery of novel RNA-based therapeutics.

Keywords:
RNA:protein interactionsRNA‐degraderRNA‐targetingbioactive small moleculescomputer‐aided designmachine learning

More Related Videos

High-throughput Screening for Broad-spectrum Chemical Inhibitors of RNA Viruses
11:34

High-throughput Screening for Broad-spectrum Chemical Inhibitors of RNA Viruses

Published on: May 5, 2014

13.9K
Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
12:03

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy

Published on: September 5, 2016

8.0K

Related Experiment Videos

Last Updated: Sep 10, 2025

Using In Vitro and In-cell SHAPE to Investigate Small Molecule Induced Pre-mRNA Structural Changes
11:58

Using In Vitro and In-cell SHAPE to Investigate Small Molecule Induced Pre-mRNA Structural Changes

Published on: January 30, 2019

8.4K
High-throughput Screening for Broad-spectrum Chemical Inhibitors of RNA Viruses
11:34

High-throughput Screening for Broad-spectrum Chemical Inhibitors of RNA Viruses

Published on: May 5, 2014

13.9K
Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
12:03

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy

Published on: September 5, 2016

8.0K

Area of Science:

  • Drug discovery and development
  • Molecular biology
  • Medicinal chemistry

Background:

  • RNA-targeting small molecules represent a significant advancement in drug discovery.
  • They offer potential treatments for diseases previously considered undruggable.
  • This field is rapidly evolving with new methodologies and strategies.

Purpose of the Study:

  • To review recent progress in the development of RNA-binding small molecules.
  • To identify current challenges and future research directions.
  • To highlight the role of AI and machine learning in this domain.

Main Methods:

  • Innovations in RNA structure determination (X-ray crystallography, NMR, cryo-EM).
  • Computational approaches including deep learning and molecular docking.
  • Screening techniques like focused libraries, DNA-encoded libraries, and fragment-based drug discovery.

Main Results:

  • Significant progress in rational drug design based on RNA structure.
  • Enhanced efficiency in RNA structure prediction and ligand screening via computational tools.
  • Emerging strategies like RNA degraders and RNA-protein interaction modulators show therapeutic promise.

Conclusions:

  • Artificial intelligence and machine learning are crucial for accelerating RNA-targeted therapeutic discovery.
  • Further research and collaboration are needed to realize the full potential of RNA-targeting small molecules.
  • These molecules could revolutionize treatment paradigms across various diseases.