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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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Small interfering RNAs (siRNA)

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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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Related Experiment Video

Updated: Jun 22, 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

[SiRNA-based therapeutics].

Motomu Shimaoka¹

¹Program of Cellular and Molecular Medicine, Children's Hospital, Boston, USA.

Nihon Rinsho. Japanese Journal of Clinical Medicine

|June 11, 2009

Summary

This summary is machine-generated.

RNAi therapeutics face delivery challenges. Novel technologies now enable targeted delivery of small interfering RNAs (siRNAs) for effective gene silencing in vivo.

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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

Published on: July 23, 2016

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Last Updated: Jun 22, 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

Area of Science:

Molecular Biology
Genetics
Biotechnology

Context:

RNA interference (RNAi) discovered in 1998, Nobel Prize awarded in 2006.
Significant challenges remain in translating RNAi into clinical treatments.
Key hurdles include targeted delivery of intact RNA molecules and avoiding off-target effects or immune responses.

Purpose:

To overcome the limitations of RNAi delivery for therapeutic applications.
To develop and evaluate novel technologies for systemic and targeted siRNA delivery.
To achieve robust and specific gene silencing in vivo.

Summary:

RNAi-based medicine development is ongoing, with delivery being a major obstacle.
New technologies facilitate the systemic and targeted delivery of small interfering RNAs (siRNAs).
These advancements enable effective in vivo gene silencing with reduced side effects.

Impact:

Potential for new RNAi-based therapies.
Improved methods for delivering therapeutic RNAs.
Advancement of gene silencing strategies for clinical use.