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siRNA - Small Interfering RNAs

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 RNAi

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...

CRISPR

CRISPR

Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced Short...

RNA Interference

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...

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Related Experiment Video

Updated: Jul 3, 2026

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery

Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery

Published on: May 2, 2019

Chemically modified siRNA: tools and applications.

Jonathan K Watts¹, Glen F Deleavey, Masad J Damha

¹Department of Chemistry, McGill University, 801 Sherbrooke Street West, Montreal, QC H3A 2K6, Canada. jonathan.watts@mail.mcgill.ca

Drug Discovery Today

|July 11, 2008

Summary

This summary is machine-generated.

Chemical modification of small interfering RNA (siRNA) enhances therapeutic potential by improving specificity and delivery. This review covers siRNA modifications, applications, and benefits like increased stability and potency.

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Area of Science:

Biochemistry
Molecular Biology
Pharmacology

Background:

Small interfering RNA (siRNA) therapeutics face challenges including delivery, off-target effects, and nuclease degradation.
Chemical modifications offer a promising strategy to overcome these limitations.

Purpose of the Study:

To review various chemical modifications of siRNA.
To examine the impact of these modifications on RNA structure and duplex architecture.
To discuss applications focusing on specificity and delivery, and improvements in nuclease stability and potency.

Main Methods:

Comprehensive literature review of siRNA chemical modifications.
Analysis of structural aspects of modified siRNA.
Evaluation of application-focused studies on specificity, delivery, stability, and potency.

Main Results:

Diverse chemical modifications exist for siRNA, impacting structure and function.
Modifications enhance specificity by reducing immune responses and off-target hybridization.
Improved nuclease stability and delivery efficiency are key outcomes of chemical modification.

Conclusions:

Chemical modification is crucial for advancing siRNA therapeutics.
Optimized siRNA modifications can lead to safer and more effective gene silencing therapies.
Further research into novel modifications will expand therapeutic applications.