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

Targeted Cancer Therapies02:57

Targeted Cancer Therapies

7.8K
The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
7.8K
Transducer Mechanism: Enzyme-Linked Receptors01:27

Transducer Mechanism: Enzyme-Linked Receptors

2.6K
Enzyme-linked receptors are cell-surface receptors acting as an enzyme or associating with an enzyme intracellularly. They make excellent drug targets. Drugs can bind to the extracellular ligand-binding domain or directly affect their enzymatic domain and alter their activity.
Major types that are helpful drug targets include:
2.6K
Transducer Mechanism: Nuclear Receptors01:31

Transducer Mechanism: Nuclear Receptors

1.4K
Nuclear receptors, or NRs, are unique transcription factors that regulate gene transcription and affect the cellular pathways involved in reproduction, development, or metabolism. Their ability to be stimulated by small lipophilic ligands and control vital cellular processes makes them ideal drug targets. Nearly 10-15% of currently prescribed drugs target these receptors.
About 48 different soluble family members of nuclear receptors are identified that can be divided into two main classes:
1.4K
Targets for Drug Action: Overview01:26

Targets for Drug Action: Overview

6.7K
Drugs target macromolecules to modify ongoing cellular processes. Primary drug targets include receptors, ion channels, transporters, and enzymes.
Receptors are either membrane-spanning or intracellular proteins, which upon binding a ligand, get activated and transmit the signal downstream to elicit a response. Drugs bind receptors, either mimicking the action of endogenous ligands or blocking the receptor activity to bring about a modified response. Nearly 35% of approved drugs target the G...
6.7K
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

99
Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
99
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

You might also read

Related Articles

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

Sort by
Same author

MYC-Mediated USP39 Upregulation Stabilizes SRSF1 in Pancreatic Cancer.

Molecular cancer research : MCR·2026
Same author

ASO-based PKM splice-switching therapy increases anti-CTLA-4 antibody efficacy in pancreatic ductal adenocarcinoma.

Cell discovery·2026
Same author

Exon-Skipping Antisense Oligonucleotides for H3.3K27M-Altered Diffuse Midline Glioma Therapy.

bioRxiv : the preprint server for biology·2026
Same author

Targeting <i>EZH2</i> Oncogenic Splicing: Decoding the Regulatory Network and Antisense Correction.

bioRxiv : the preprint server for biology·2026
Same author

Splice-switching ASOs targeting the AURKA 5' UTR collapse an SRSF1-AURKA-MYC oncogenic circuit in pancreatic cancer.

Molecular cell·2025
Same author

Modulating NLRP3 splicing with antisense oligonucleotides to control pathological inflammation.

Nucleic acids research·2025
Same journal

Optimized tRNA structure-seq reveals robust tRNA secondary structures in <i>S. cerevisiae</i> under mild stress conditions.

RNA (New York, N.Y.)·2026
Same journal

SERIPH: A Two-Step Extraction Protocol for Selective Enrichment of Semi-Extractable RNAs.

RNA (New York, N.Y.)·2026
Same journal

Reduced Sensitivity to RNA Structural Differences Distinguishes Eukaryotic Pus4 from Bacterial TruB.

RNA (New York, N.Y.)·2026
Same journal

Puf3 contributes to changes in mRNA solubility, translation elongation dynamics at rare arginine codons and loss of protein homeostasis in cells lacking Not4.

RNA (New York, N.Y.)·2026
Same journal

RBM38 Regulates HORMAD1 Splicing to Enhances MEK Inhibitor Sensitivity in Breast Cancer.

RNA (New York, N.Y.)·2026
Same journal

EF-P Inhibits Ribosomal α-Hydroxy Acid Incorporation: Strategic tRNA Body Selection for Co-incorporating α-Hydroxy Acids and Nonproteinogenic Amino Acids into Depsipeptides.

RNA (New York, N.Y.)·2026
See all related articles

Related Experiment Video

Updated: Aug 6, 2025

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

7.9K

RNA therapeutics.

Michelle L Hastings1, Adrian R Krainer2

  • 1Rosalind Franklin University of Medicine and Science, Chicago, Illinois 60064, USA.

RNA (New York, N.Y.)
|March 17, 2023
PubMed
Summary
This summary is machine-generated.

RNA therapeutics offer novel disease treatments by using RNA as a drug or target. This field is rapidly advancing, presenting new opportunities for drug development and therapeutic strategies.

More Related Videos

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
09:37

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells

Published on: August 25, 2021

1.8K
Analyzing Tumor and Tissue Distribution of Target Antigen Specific Therapeutic Antibody
07:36

Analyzing Tumor and Tissue Distribution of Target Antigen Specific Therapeutic Antibody

Published on: May 16, 2020

5.4K

Related Experiment Videos

Last Updated: Aug 6, 2025

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

7.9K
Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
09:37

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells

Published on: August 25, 2021

1.8K
Analyzing Tumor and Tissue Distribution of Target Antigen Specific Therapeutic Antibody
07:36

Analyzing Tumor and Tissue Distribution of Target Antigen Specific Therapeutic Antibody

Published on: May 16, 2020

5.4K

Area of Science:

  • Biochemistry
  • Molecular Biology
  • Pharmacology

Background:

  • RNA therapeutics leverage ribonucleic acid (RNA) for disease treatment, either as a drug itself or as a target for small-molecule drugs.
  • Recent advancements in technology and clinical successes have significantly improved RNA's drug-like properties and its viability as a therapeutic target.
  • The field is experiencing a renaissance, driven by breakthroughs that enhance RNA's potential in medicine.

Purpose of the Study:

  • To provide a comprehensive overview of RNA therapeutics, covering historical context, current applications, and future directions.
  • To serve as an introductory guide for researchers new to the field of RNA therapeutics.
  • To encourage broader scientific engagement with RNA-based therapeutic strategies and drug development.

Main Methods:

  • This special issue compiles expert perspectives and reviews on the field of RNA therapeutics.
  • It synthesizes current knowledge on RNA's role as both a therapeutic agent and a drug target.
  • The content is designed to inform and inspire the scientific community.

Main Results:

  • Expert insights highlight the transformative potential of RNA in drug development.
  • The review underscores the expanding utility of RNA-based therapies across various diseases.
  • Breakthrough technologies are enhancing the efficacy and applicability of RNA therapeutics.

Conclusions:

  • RNA therapeutics represent a rapidly evolving and promising area of medical research and drug development.
  • The field challenges the scientific community to integrate RNA into experimental designs and therapeutic strategies.
  • Harnessing the full potential of RNA is crucial for future innovations in treating diseases.