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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...

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Related Experiment Video

Updated: Jun 6, 2026

Long-term Silencing of Intersectin-1s in Mouse Lungs by Repeated Delivery of a Specific siRNA via Cationic Liposomes. Evaluation of Knockdown Effects by Electron Microscopy

Long-term Silencing of Intersectin-1s in Mouse Lungs by Repeated Delivery of a Specific siRNA via Cationic Liposomes. Evaluation of Knockdown Effects by Electron Microscopy

Published on: June 21, 2013

Cationic nucleolipids as efficient siRNA carriers.

Hye Won Yang¹, Jeong Wu Yi, Eun-Kyoung Bang

¹Department of Chemistry, BK School of Molecular Science, Pohang University of Science and Technology, Pohang, 790-784 Korea.

Organic & Biomolecular Chemistry

|November 18, 2010

Summary

This summary is machine-generated.

Researchers developed novel uridine-based cationic nucleolipids for efficient siRNA delivery. These compounds showed effective in vitro cellular delivery with minimal toxicity, offering a promising gene silencing approach.

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Published on: February 1, 2019

Area of Science:

Medicinal Chemistry
Biotechnology
Nucleoside Analog Development

Background:

Developing effective delivery systems for small interfering RNA (siRNA) is crucial for gene therapy.
Nucleolipids offer potential as non-viral vectors due to their biocompatibility and tunable properties.

Purpose of the Study:

To synthesize and characterize novel uridine-based cationic nucleolipids.
To evaluate the efficacy and safety of these nucleolipids as siRNA delivery vehicles.

Main Methods:

Synthesis of five novel uridine-based cationic nucleolipids via 1,3-dipolar cycloaddition and carbamate linkages.
Formation of lipoplexes with siRNA.
In vitro evaluation of cellular uptake and cytotoxicity.

Main Results:

Successful synthesis of five distinct uridine-based cationic nucleolipids.
Efficient delivery of siRNA into cells mediated by the synthesized nucleolipid lipoplexes.
Demonstrated lack of severe toxicity in vitro.

Conclusions:

Novel uridine-based cationic nucleolipids are effective siRNA delivery agents.
These nucleolipids represent a promising platform for gene silencing applications.
The developed compounds exhibit a favorable safety profile for in vitro use.