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

RNA Interference01:23

RNA Interference

26.2K
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...
26.2K
siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

16.9K
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...
16.9K
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
Small interfering RNAs (siRNA)02:30

Small interfering RNAs (siRNA)

3.6K
3.6K
Types of RNA01:23

Types of RNA

64.2K
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...
64.2K
Translational Regulation01:29

Translational Regulation

47
Translational regulation in prokaryotes ensures efficient protein synthesis by controlling ribosome access to mRNA. This regulation is mediated by secondary RNA structures, including translational riboswitches, RNA thermometers, and small RNAs (sRNAs), which respond to intracellular and environmental signals to modulate gene expression.Translational RiboswitchesRiboswitches in the leader region of mRNAs can regulate translation by altering the accessibility of the Shine-Dalgarno (SD) sequence,...
47

You might also read

Related Articles

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

Sort by
Same author

Statistical versus clinical significance: what is the (clinically important) difference?

Drug and therapeutics bulletin·2026
Same journal

Pharmacological treatment of polyendocrine metabolic ovarian syndrome in adults.

Drug and therapeutics bulletin·2026
Same journal

Lipid-lowering for people with chronic kidney disease: a narrative review.

Drug and therapeutics bulletin·2026
Same journal

Study confirms benefits of continuous glucose monitoring in type 2 diabetes.

Drug and therapeutics bulletin·2026
Same journal

Update on spasticity: part-2 - physical, pharmacological and surgical management.

Drug and therapeutics bulletin·2026
Same journal

Reducing thrombosis risk much earlier in pregnancy: the new NHS Maternal Care Bundle.

Drug and therapeutics bulletin·2026
Same journal

Once-weekly insulin (insulin efsitora) for patients with type 2 diabetes.

Drug and therapeutics bulletin·2026
See all related articles

Related Experiment Video

Updated: Aug 1, 2025

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery
09:09

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery

Published on: May 2, 2019

7.5K

Small interfering RNA (siRNA)-based therapeutics.

Jacoby Patterson1

  • 1Senior Research Consultant, Windsor, UK jacobyvpatterson@gmail.com.

Drug and Therapeutics Bulletin
|April 25, 2023
PubMed
Summary
This summary is machine-generated.

Small interfering RNA (siRNA) therapies silence specific genes by degrading messenger RNA (mRNA), reducing harmful protein production. This gene silencing approach is effective for genetic conditions and is advancing in clinical development.

Keywords:
Evidence-Based MedicineHypercholesterolemiaTherapeutics

More Related Videos

Nanoparticle-mediated siRNA Gene-silencing in Adult Zebrafish Heart
09:12

Nanoparticle-mediated siRNA Gene-silencing in Adult Zebrafish Heart

Published on: July 29, 2018

8.2K
MISSION esiRNA for RNAi Screening in Mammalian Cells
15:31

MISSION esiRNA for RNAi Screening in Mammalian Cells

Published on: May 12, 2010

16.4K

Related Experiment Videos

Last Updated: Aug 1, 2025

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery
09:09

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery

Published on: May 2, 2019

7.5K
Nanoparticle-mediated siRNA Gene-silencing in Adult Zebrafish Heart
09:12

Nanoparticle-mediated siRNA Gene-silencing in Adult Zebrafish Heart

Published on: July 29, 2018

8.2K
MISSION esiRNA for RNAi Screening in Mammalian Cells
15:31

MISSION esiRNA for RNAi Screening in Mammalian Cells

Published on: May 12, 2010

16.4K

Area of Science:

  • Molecular Biology
  • Genetics
  • Pharmacology

Background:

  • Small interfering RNA (siRNA) molecules experimentally cleave intracellular messenger RNA (mRNA).
  • This process reduces protein synthesis, effectively 'silencing' specific genes.
  • Early studies demonstrated siRNA's potential in simple organisms and mammalian cell lines.

Purpose of the Study:

  • To assess the therapeutic effects of siRNA in patients with genetic conditions.
  • To evaluate siRNA's ability to reduce excessive harmful protein levels, such as amyloid.
  • To explore delivery methods for enhancing siRNA efficacy and specificity.

Main Methods:

  • Formulation of hydrophilic siRNA molecules into lipid nanoparticles for cellular transport.
  • Conjugation of siRNA to targeting molecules for specific cell action (e.g., hepatocytes).
  • Clinical assessment of siRNA in patients with genetic disorders.

Main Results:

  • siRNA demonstrated the ability to reduce harmful protein production in patients.
  • Intracellular effects of siRNA can persist for several months.
  • siRNA exhibits high specificity due to exact complementary sequence requirements.

Conclusions:

  • siRNA technology offers a promising therapeutic strategy for genetic diseases.
  • Several siRNA-based medicines are licensed, with numerous others in development.
  • Potential applications span genetic hepatic, cardiovascular, and ocular conditions.