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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...

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...

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...

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...

Inhibitors of Viral Protein Synthesis

Inhibitors of Viral Protein Synthesis

Protein synthesis is indispensable for viral replication, as viruses lack the cellular machinery required for this process and must hijack the host's translational apparatus. In response, host cells deploy a critical innate immune defense involving interferons, specialized cytokines that play a central role in inhibiting viral propagation.Upon viral detection, infected cells release interferons that bind to receptors on adjacent uninfected cells, activating the JAK-STAT signaling pathway and...

Homologous Recombination

Homologous Recombination

The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...

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Related Experiment Video

Updated: Jun 20, 2026

Dual CRISPR-Interference Strategy for Targeting Synthetic Lethal Interactions Between Non-Coding RNAs in Cancer Cells

Dual CRISPR-Interference Strategy for Targeting Synthetic Lethal Interactions Between Non-Coding RNAs in Cancer Cells

Published on: May 30, 2025

RNA interference technologies and therapeutics: from basic research to products.

Marta López-Fraga¹, Tamara Martínez, Ana Jiménez

¹Sylentis SAU, Parque Tecnológico de Madrid, Madrid, Spain. mlfraga33@gmail.com

Biodrugs : Clinical Immunotherapeutics, Biopharmaceuticals and Gene Therapy

|September 17, 2009

Summary

This summary is machine-generated.

RNA interference (RNAi) offers a powerful therapeutic approach by silencing disease-causing genes. Advances in delivery systems are crucial for effective clinical applications of RNAi therapies.

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Area of Science:

Molecular Biology
Genetics
Pharmacology

Background:

RNA interference (RNAi) is a natural gene silencing mechanism.
RNAi therapeutics can target previously undruggable disease-associated genes.
The potential of RNAi spans various diseases and cell types.

Purpose of the Study:

To review current knowledge of RNAi mechanisms.
To discuss safety considerations for therapeutic RNAi.
To highlight advancements in RNAi delivery systems and clinical applications.

Main Methods:

Review of scientific literature on RNAi mechanisms.
Analysis of safety profiles and efficacy of RNAi therapeutics.
Examination of viral and non-viral delivery systems for RNAi.
Discussion of challenges in clinical translation.

Main Results:

RNAi provides a versatile platform for targeting diverse pathologies.
Optimized nucleic acid design and chemical modifications enhance stability and efficacy.
Development of sophisticated delivery systems is key for targeted delivery and reduced dosage.
Ongoing preclinical and clinical studies demonstrate therapeutic potential.

Conclusions:

RNAi technology holds significant promise for treating genetic, infectious, and other diseases.
Overcoming challenges in delivery, safety, and regulatory pathways is essential for clinical success.
Continued research in RNAi mechanisms and delivery systems will drive therapeutic innovation.