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

MicroRNAs01:22

MicroRNAs

3.0K
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...
3.0K
MicroRNAs01:22

MicroRNAs

20.6K
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...
20.6K
MicroRNAs01:22

MicroRNAs

9.7K
9.7K
Experimental RNAi02:15

Experimental RNAi

6.5K
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...
6.5K
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

1.3K
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.
1.3K
RNA Interference01:23

RNA Interference

24.0K
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...
24.0K

You might also read

Related Articles

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

Sort by
Same author

Regulation of fibroblast growth factor 2 expression in melanoma cells by the c-MYB proto-oncoprotein.

Cell growth & differentiation : the molecular biology journal of the American Association for Cancer Research·1998
Same author

Characterization of a polymorphism in NAD(P)H: quinone oxidoreductase (DT-diaphorase).

British journal of cancer·1997
Same author

Role of NAD(P)H:quinone oxidoreductase (DT-diaphorase) in cytotoxicity and induction of DNA damage by streptonigrin.

Biochemical pharmacology·1996
Same author

Reactive oxygen species and DNA damage in 2-bromo-(glutathion-S-yl) hydroquinone-mediated cytotoxicity.

Archives of biochemistry and biophysics·1995
Same author

Increased activity and expression of NAD(P)H:quinone acceptor oxidoreductase in confluent cell cultures and within multicellular spheroids.

Cancer research·1994
Same author

Metabolism of bioreductive antitumor compounds by purified rat and human DT-diaphorases.

Cancer research·1994
Same journal

Dr Archibald Hewan (1832-1883) and the University of Glasgow's medical libraries.

The journal of the Royal College of Physicians of Edinburgh·2025
Same journal

Joyce Grainger Learning Centre.

The journal of the Royal College of Physicians of Edinburgh·2025
Same journal

Features in this issue.

The journal of the Royal College of Physicians of Edinburgh·2025
Same journal

An unusual case of cutaneous tuberculosis and paradoxical TB in a patient with spondyloarthritis treated with adalimumab.

The journal of the Royal College of Physicians of Edinburgh·2025
Same journal

Leptospiral infection overlapping with autoimmune hepatitis: A diagnostic conundrum.

The journal of the Royal College of Physicians of Edinburgh·2025
Same journal

Tocilizumab vs bevacizumab in critically ill COVID-19 patients: Registry-based prospective study.

The journal of the Royal College of Physicians of Edinburgh·2025
See all related articles

Related Experiment Video

Updated: Apr 22, 2026

In Vivo Nanovector Delivery of a Heart-specific MicroRNA-sponge
09:53

In Vivo Nanovector Delivery of a Heart-specific MicroRNA-sponge

Published on: June 15, 2018

7.2K

Engineered microRNA therapeutics.

N W Gibson1

  • 1NW Gibson, Regulus Therapeutics Inc, 3545 John Hopkins Court, San Diego, CA 92121-1121, USA. Email ngibson@regulusrx.com.

The Journal of the Royal College of Physicians of Edinburgh
|October 17, 2014
PubMed
Summary
This summary is machine-generated.

Engineered microRNA therapeutics offer novel disease treatment strategies by targeting microRNA dysregulation. These approaches, including antimiRs and microRNA mimics, are advancing into clinical trials for various conditions.

Keywords:
anti-miR therapeuticsdeliveryengineered microRNA therapeuticsmicroRNA mimicsoligonucleotides

More Related Videos

Characterization of Functionally Associated miRNAs in Glioblastoma and their Engineering into Artificial Clusters for Gene Therapy
09:40

Characterization of Functionally Associated miRNAs in Glioblastoma and their Engineering into Artificial Clusters for Gene Therapy

Published on: October 4, 2019

5.2K
MicroRNA Amplification and Recognition through Locked-nucleic-acid In situ Hybridization as a Novel Detection and Quantification Method
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

547

Related Experiment Videos

Last Updated: Apr 22, 2026

In Vivo Nanovector Delivery of a Heart-specific MicroRNA-sponge
09:53

In Vivo Nanovector Delivery of a Heart-specific MicroRNA-sponge

Published on: June 15, 2018

7.2K
Characterization of Functionally Associated miRNAs in Glioblastoma and their Engineering into Artificial Clusters for Gene Therapy
09:40

Characterization of Functionally Associated miRNAs in Glioblastoma and their Engineering into Artificial Clusters for Gene Therapy

Published on: October 4, 2019

5.2K
MicroRNA Amplification and Recognition through Locked-nucleic-acid In situ Hybridization as a Novel Detection and Quantification Method
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

547

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Pharmacology

Background:

  • MicroRNAs (miRNAs) play crucial roles in gene regulation and are implicated in various diseases.
  • Dysregulation of miRNA expression, either overexpression or loss, drives disease progression.
  • Current therapeutic strategies often target individual proteins, missing opportunities to modulate complex disease pathways.

Purpose of the Study:

  • To explore novel therapeutic strategies targeting microRNA dysregulation.
  • To introduce engineered microRNA therapeutics, including antimiRs and miRNA mimics, as potential treatments.
  • To highlight the potential of modulating multiple disease-related proteins via miRNA therapeutics.

Main Methods:

  • Development of chemically modified single-stranded oligonucleotides (antimiRs) to inhibit overexpressed miRNAs.
  • Design of double-stranded nucleic acid molecules (miRNA mimics) to restore lost miRNA expression.
  • Advancement of engineered miRNA therapeutics into clinical development.

Main Results:

  • Engineered miRNA therapeutics demonstrate potential for treating diseases driven by miRNA dysregulation.
  • Human proof-of-concept achieved with an antimiR targeting miR-122, crucial for Hepatitis C virus replication.
  • The approach allows for the modulation of multiple proteins involved in disease pathogenesis.

Conclusions:

  • Engineered microRNA therapeutics represent a paradigm shift in disease treatment.
  • AntimiRs and miRNA mimics offer distinct yet complementary approaches to address miRNA dysregulation.
  • These novel therapeutics hold promise for revolutionizing disease management by targeting complex molecular networks.