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

6.1K
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...
6.1K
Structure-Activity Relationships and Drug Design01:28

Structure-Activity Relationships and Drug Design

716
Drug design is a dynamic field that involves discovering and developing new medications based on specific biological targets. This process heavily relies on structure-activity relationships (SAR) and quantitative structure-activity relationships (QSAR) to guide the design and optimization of efficient drugs.
SAR studies the intricate relationship between a drug's chemical structure and biological activity. It focuses on understanding how modifications to a drug's structure can influence...
716
siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

16.8K
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...
16.8K
Drug Discovery: Overview01:26

Drug Discovery: Overview

7.9K
Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
7.9K
Types of RNA01:23

Types of RNA

63.6K
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...
63.6K
RNA Editing02:23

RNA Editing

9.0K
RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
9.0K

You might also read

Related Articles

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

Sort by
Same author

Mismatch repair dissection by in vivo RNAi reveals dose-dependent modulators of somatic instability and proteome remodeling in Huntington's disease.

bioRxiv : the preprint server for biology·2026
Same author

Precision RNAi for Fibrodysplasia Ossificans Progressiva: a combinatorial, unimolecular, allele selective approach.

Research square·2026
Same author

Fully Modified SpyCas9 Guide RNAs Enable Robust Genome Editing In Cells and In Vivo.

bioRxiv : the preprint server for biology·2026
Same author

C57BL/6 BAC-CAG Huntington's disease mice show somatic CAG expansion and responses to small interfering RNAs comparable to the FVB strain.

bioRxiv : the preprint server for biology·2026
Same author

Divalent siRNA for prion disease.

Nucleic acids research·2026
Same author

Lowering the <i>HTT1a</i> transcript as an effective therapy for Huntington's disease in a knockin mouse model.

Science translational medicine·2026

Related Experiment Video

Updated: Jun 29, 2025

An Optimized Quantitative Pull-Down Analysis of RNA-Binding Proteins Using Short Biotinylated RNA
07:55

An Optimized Quantitative Pull-Down Analysis of RNA-Binding Proteins Using Short Biotinylated RNA

Published on: February 17, 2023

3.6K

RNAi-based drug design: considerations and future directions.

Qi Tang1,2, Anastasia Khvorova3,4

  • 1RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, USA.

Nature Reviews. Drug Discovery
|April 3, 2024
PubMed
Summary

RNA interference (RNAi) therapies, using small interfering RNA (siRNA), show promise for drug development. Overcoming delivery challenges is key to expanding siRNA therapeutics beyond the liver for various diseases.

More Related Videos

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

8.3K
Quantitative Structure-Activity Relationship, Activity Prediction, and Molecular Dynamics of Non-nucleotide Reverse Transcriptase Inhibitors
10:29

Quantitative Structure-Activity Relationship, Activity Prediction, and Molecular Dynamics of Non-nucleotide Reverse Transcriptase Inhibitors

Published on: May 9, 2025

1000

Related Experiment Videos

Last Updated: Jun 29, 2025

An Optimized Quantitative Pull-Down Analysis of RNA-Binding Proteins Using Short Biotinylated RNA
07:55

An Optimized Quantitative Pull-Down Analysis of RNA-Binding Proteins Using Short Biotinylated RNA

Published on: February 17, 2023

3.6K
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

8.3K
Quantitative Structure-Activity Relationship, Activity Prediction, and Molecular Dynamics of Non-nucleotide Reverse Transcriptase Inhibitors
10:29

Quantitative Structure-Activity Relationship, Activity Prediction, and Molecular Dynamics of Non-nucleotide Reverse Transcriptase Inhibitors

Published on: May 9, 2025

1000

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Pharmaceutical Sciences

Background:

  • RNA interference (RNAi) is a gene regulation mechanism with significant therapeutic potential.
  • Recent FDA approvals highlight the success of small interfering RNA (siRNA) drugs targeting the liver.
  • Significant challenges, particularly in delivery, hinder the broader application of siRNA therapeutics.

Purpose of the Study:

  • To review key considerations and future directions in designing therapeutic siRNAs.
  • To emphasize the role of chemistry, informatics, and delivery strategies in successful siRNA drug development.
  • To discuss the importance of target selection for optimizing therapeutic outcomes.

Main Methods:

  • Review of current literature on RNAi therapeutics and drug development.
  • Analysis of chemical modifications, informatics applications, and delivery systems for siRNAs.
  • Discussion of target selection criteria and their impact on therapeutic efficacy.

Main Results:

  • Synthetic siRNAs offer potent and durable gene silencing capabilities.
  • Current siRNA drugs are primarily approved for hepatic targets, limiting extrahepatic applications.
  • Delivery remains a critical bottleneck for advancing siRNA therapies to non-liver tissues.

Conclusions:

  • Continued innovation in siRNA design, chemistry, and delivery is essential.
  • Integrating informatics and careful target selection can enhance therapeutic success.
  • Expanding the utility of siRNA therapeutics beyond the liver requires addressing current limitations.