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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.
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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 Interference

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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.
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RNA Interference

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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.
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Related Experiment Video

Updated: Jun 14, 2026

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System

Published on: October 29, 2019

RNAi therapeutics for CNS disorders.

Ryan L Boudreau¹, Beverly L Davidson

¹Department of Internal Medicine, University of Iowa, Iowa City, IA, USA.

|March 24, 2010

Summary

RNA interference (RNAi) offers gene silencing for disease treatment. RNAi therapeutics show promise for neurological disorders, with research advancing toward clinical trials.

Area of Science:

Molecular Biology
Genetics
Neuroscience

Background:

RNA interference (RNAi) is a natural gene silencing mechanism.
RNAi is a valuable tool for studying gene function and validating drug targets.
RNAi-based compounds are being explored as novel therapeutics for various diseases.

Purpose of the Study:

To review RNAi-based gene-silencing strategies for neurological disorders.
To examine proof-of-concept studies of therapeutic RNAi in CNS disorders.
To highlight recent advancements in transitioning RNAi therapeutics to clinical trials.

Main Methods:

Review of existing literature on RNAi technology and its therapeutic applications.
Analysis of studies demonstrating RNAi efficacy in preclinical models of neurological diseases.

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Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes

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Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice

Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice

Published on: February 24, 2026

Related Experiment Videos

Last Updated: Jun 14, 2026

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System

Intrathecal Delivery of Antisense Oligonucleotides in the Rat Central Nervous System

Published on: October 29, 2019

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes

Published on: July 23, 2016

Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice

Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice

Published on: February 24, 2026

Focus on gene-silencing strategies targeting mutant or toxic gene expression.

Main Results:

RNAi therapeutics have shown potential in improving disease phenotypes in rodent models.
Reduction of mutant or toxic gene expression is a key therapeutic strategy for CNS disorders.
Significant progress has been made in developing RNAi-based treatments for neurological conditions.

Conclusions:

RNAi-based therapies hold significant promise for treating neurological disorders.
Further research and development are crucial for clinical translation of RNAi therapeutics.
The field is moving towards clinical trials for RNAi-based treatments for CNS diseases.