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Related Concept Videos

siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

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 ATP-dependent...
Experimental RNAi02:15

Experimental RNAi

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...
RNA Interference01:23

RNA Interference

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...
RNA Interference01:23

RNA Interference

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...
piRNA - Piwi-interacting RNAs02:57

piRNA - Piwi-interacting RNAs

PIWI-interacting RNAs, or piRNAs, are the most abundant short non-coding RNAs. More than 20,000 genes have been found in humans that code for piRNAs while only 2000 genes have been found for miRNAs. piRNAs can act at the transcriptional and post-transcriptional levels and have a vital role in silencing transposable elements present in germ cells. They are also involved in epigenetic silencing and activation. Previously, they were thought to function only in germ cells but new evidence suggests...
lncRNA - Long Non-coding RNAs02:39

lncRNA - Long Non-coding RNAs

In humans, more than 80% of the genome gets transcribed. However, only around 2% of the genome codes for proteins. The remaining part produces non-coding RNAs which includes ribosomal RNAs, transfer RNAs, telomerase RNAs, and regulatory RNAs, among other types. A large number of regulatory non-coding RNAs have been classified into two groups depending upon their length – small non-coding RNAs, such as microRNA, which are less than 200 nucleotides in length, and long non-coding RNA (lncRNA)...

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Related Experiment Video

Updated: May 19, 2026

Rearing and Double-stranded RNA-mediated Gene Knockdown in the Hide Beetle, Dermestes maculatus
09:57

Rearing and Double-stranded RNA-mediated Gene Knockdown in the Hide Beetle, Dermestes maculatus

Published on: December 28, 2016

Single-stranded siRNAs activate RNAi in animals.

Walt F Lima1, Thazha P Prakash, Heather M Murray

  • 1Core Antisense Research, Isis Pharmaceuticals Inc., Carlsbad, CA 92010, USA.

Cell
|September 4, 2012
PubMed
Summary
This summary is machine-generated.

Potent single-stranded siRNAs (ss-siRNAs) can silence genes in vivo without complex delivery. This discovery simplifies RNA interference (RNAi) therapeutics and offers new insights into RNAi mechanisms.

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Cell Based Assays of SINEUP Non-coding RNAs That Can Specifically Enhance mRNA Translation
10:21

Cell Based Assays of SINEUP Non-coding RNAs That Can Specifically Enhance mRNA Translation

Published on: February 1, 2019

Related Experiment Videos

Last Updated: May 19, 2026

Rearing and Double-stranded RNA-mediated Gene Knockdown in the Hide Beetle, Dermestes maculatus
09:57

Rearing and Double-stranded RNA-mediated Gene Knockdown in the Hide Beetle, Dermestes maculatus

Published on: December 28, 2016

Cell Based Assays of SINEUP Non-coding RNAs That Can Specifically Enhance mRNA Translation
10:21

Cell Based Assays of SINEUP Non-coding RNAs That Can Specifically Enhance mRNA Translation

Published on: February 1, 2019

Area of Science:

  • Biochemistry
  • Molecular Biology
  • Pharmacology

Background:

  • Therapeutic applications of small interfering RNAs (siRNAs) are hindered by the need for complex delivery systems.
  • Identifying potent single-stranded RNAs could streamline the development of RNA-based drugs.

Purpose of the Study:

  • To develop and characterize single-stranded siRNAs (ss-siRNAs) capable of gene silencing in vivo without lipid formulations.
  • To explore the structure-activity relationships of ss-siRNAs for enhanced potency, stability, and pharmacokinetic properties.

Main Methods:

  • Iterative design and chemical modification of single-stranded RNAs.
  • Structure-activity relationship studies correlating chemical modifications with Argonaute 2 (AGO2) activity, cellular potency, nuclease stability, and pharmacokinetics.
  • In vivo testing of ss-siRNAs for gene silencing efficacy.

Main Results:

  • Identified effective ss-siRNAs that silence gene expression in animals without lipid-based delivery.
  • Demonstrated that the passenger strand is not essential for potent gene silencing via the RNA interference (RNAi) pathway.
  • Confirmed that ss-siRNA activity in vivo requires a 5' phosphate, leading to the development of a stable 5'-(E)-vinylphosphonate (5'-VP) analog.

Conclusions:

  • Potent ss-siRNAs represent a simplified approach to RNAi therapeutics, bypassing complex delivery requirements.
  • The findings provide a new perspective on the mechanism of RNAi, highlighting the essential role of the guide strand and 5' phosphate.
  • This work expands the options for developing RNAi-based pharmacological agents.