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Related Concept Videos

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...
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...
Ribosome Profiling02:24

Ribosome Profiling

Ribosome profiling or ribo-sequencing is a deep sequencing technique that produces a snapshot of active translation in a cell. It selectively sequences the mRNAs protected by ribosomes to get an insight into a cell’s translation landscape at any given point in time.
Applications of ribosome profiling
Ribosome profiling has many applications, including in vivo monitoring of translation inside a particular organ or tissue type and quantifying new protein synthesis levels.
The technique helps...
Types of RNA01:20

Types of RNA

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 regulating 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 Performs Diverse...
Riboswitches01:56

Riboswitches

Riboswitches are non-coding mRNA domains that regulate the transcription and translation of downstream genes without the help of proteins. Riboswitches bind directly to a metabolite and can form unique stem-loop or hairpin structures in response to the amount of the metabolite present. They have two distinct regions – a metabolite-binding aptamer and an expression platform.
The aptamer has high specificity for a particular metabolite which allows riboswitches to specifically regulate...

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Related Experiment Video

Updated: Jun 11, 2026

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

A status report on RNAi therapeutics.

Akshay K Vaishnaw1, Jared Gollob, Christina Gamba-Vitalo

  • 1Alnylam Pharmaceuticals Inc,, 300 Third Street, Cambridge, MA 02142, USA. avaishnaw@alnylam.com.

Silence
|July 10, 2010
PubMed
Summary

RNA interference (RNAi) therapeutics, using small interfering RNAs (siRNAs), offer a potent and specific method for gene silencing. This approach holds promise for treating diseases by targeting previously undruggable proteins.

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Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System
07:47

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System

Published on: October 29, 2019

Related Experiment Videos

Last Updated: Jun 11, 2026

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

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System
07:47

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System

Published on: October 29, 2019

Area of Science:

  • Molecular Biology
  • Genetics
  • Pharmacology

Background:

  • RNA interference (RNAi) discovery in model organisms.
  • Demonstration of RNAi in mammalian cells using small interfering RNAs (siRNAs).
  • Identification of the RNA-induced silencing complex (RISC) machinery in human cells.

Purpose of the Study:

  • To review the current understanding of RNAi therapeutics.
  • To cover siRNA lead discovery, delivery, and in vivo profiles.
  • To discuss preclinical safety and clinical progress of RNAi programs.

Main Methods:

  • Review of existing literature on RNAi biology and therapeutics.
  • Analysis of siRNA lead discovery and delivery strategies.
  • Evaluation of in vivo pharmacological data and preclinical safety.

Main Results:

  • RNAi pathway offers sequence-specific and potent gene silencing.
  • siRNAs can target proteins intractable to conventional therapies.
  • 14 RNAi therapeutic programs have entered clinical trials.

Conclusions:

  • RNAi represents a significant pharmacological advance.
  • Potential to develop novel therapeutics for various diseases.
  • Ongoing clinical evaluation of RNAi-based treatments.