Related Experiment Video
Updated: May 25, 2026
09:04
Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
Published on: September 21, 2017
RNA therapeutics: beyond RNA interference and antisense oligonucleotides
Ryszard Kole1, Adrian R Krainer, Sidney Altman
1AVI BioPharma, 3450 Monte Villa Parkway, Bothell, Washington 98021, USA. rkole@avibio.com
Nature Reviews. Drug Discovery
|January 21, 2012
Summary
RNA-based therapies, including RNA interference, antisense, and steric-blocking oligonucleotides, offer new ways to treat genetic disorders. Steric-blocking oligonucleotides show promise for restoring gene function, as seen in Duchenne muscular dystrophy trials.
Area of Science:
- Biotechnology
- Molecular Biology
- Therapeutics
Background:
- RNA-based therapeutics utilize sequence-specific oligonucleotide binding to RNA.
- Different oligonucleotide types (RNA interference, antisense, steric-blocking) have distinct mechanisms and effects.
Purpose of the Study:
- To review RNA-based therapeutic technologies and their mechanisms of action.
- To highlight the potential of steric-blocking oligonucleotides for treating genetic disorders.
Main Methods:
- Discussion of three oligonucleotide-based therapeutic technologies.
- Analysis of RNA interference, antisense, and steric-blocking oligonucleotide mechanisms.
- Review of chemical modifications for improved drug-like properties.
Main Results:
- RNA interference and antisense oligonucleotides induce mRNA degradation.
- Steric-blocking oligonucleotides modulate RNA processing without degradation.
- Steric-blocking oligonucleotides can restore gene function and protein production.
Conclusions:
- Steric-blocking oligonucleotides are suitable for genetic disorder treatment due to their gene restoration capabilities.
- Clinical trials for Duchenne muscular dystrophy demonstrate the advancing potential of this technology.
Related Concept Videos
01:23RNA 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...
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...
01:23RNA 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...
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...
02:15Experimental 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...
02:30siRNA - 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...
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...
01:23Types of RNA
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...
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...
01:20Types 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...
RNA Performs Diverse...