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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...

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...

lncRNA - Long Non-coding RNAs

lncRNA - Long Non-coding RNAs

In humans, more than 80% of the genome gets transcribed. However, only around 2% of the genome codes for proteins. The remaining part produces non-coding RNAs which includes ribosomal RNAs, transfer RNAs, telomerase RNAs, and regulatory RNAs, among other types. A large number of regulatory non-coding RNAs have been classified into two groups depending upon their length – small non-coding RNAs, such as microRNA, which are less than 200 nucleotides in length, and long non-coding RNA (lncRNA)...

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...

Types of RNA

Types of RNA

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 regulating 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.
RNA Performs Diverse...

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Related Experiment Video

Updated: May 7, 2026

Testing the In Vitro and In Vivo Efficiency of mRNA-Lipid Nanoparticles Formulated by Microfluidic Mixing

Testing the In Vitro and In Vivo Efficiency of mRNA-Lipid Nanoparticles Formulated by Microfluidic Mixing

Published on: January 20, 2023

Advances in lipid-based platforms for RNAi therapeutics.

Sara Falsini¹, Laura Ciani, Sandra Ristori

¹Department of Chemistry "Ugo Schiff" and CSGI, University of Florence , Via della Lastruccia 3, 50019 Sesto Fiorentino (Fi), Italy.

Journal of Medicinal Chemistry

|September 20, 2013

Summary

RNA interference (RNAi) uses small interfering RNA (siRNA) for gene silencing, offering a promising therapeutic approach. Lipid-based vectors are crucial for effective in vivo delivery of these RNAi therapeutics, especially in clinical settings.

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Last Updated: May 7, 2026

Testing the In Vitro and In Vivo Efficiency of mRNA-Lipid Nanoparticles Formulated by Microfluidic Mixing

Testing the In Vitro and In Vivo Efficiency of mRNA-Lipid Nanoparticles Formulated by Microfluidic Mixing

Published on: January 20, 2023

Synthesizing Lipid Nanoparticles by Turbulent Flow in Confined Impinging Jet Mixers

Synthesizing Lipid Nanoparticles by Turbulent Flow in Confined Impinging Jet Mixers

Published on: August 23, 2024

Area of Science:

Molecular Medicine
Gene Therapy
Biotechnology

Background:

RNA interference (RNAi) is a natural gene silencing mechanism.
Small interfering RNA (siRNA) enables targeted gene knockdown without immune response.
RNAi therapeutics offer potential for treating diseases by controlling gene expression.

Purpose of the Study:

To discuss the application of RNAi therapeutics in molecular medicine.
To highlight the challenges and advancements in in vivo delivery systems.
To emphasize the importance of lipid-based vectors for RNAi delivery.

Main Methods:

Review of lipid-based vector formulations for RNAi delivery.
Discussion of recent clinical trials involving RNAi therapeutics.
Exploration of physicochemical characterization in RNAi therapeutic development.

Main Results:

Lipid-based vectors are the most common and biocompatible delivery systems for RNAi.
RNAi therapeutics show promise in clinical trials for conditions unresponsive to conventional treatments.
Comprehensive physicochemical characterization is vital for successful RNAi therapeutic development.

Conclusions:

RNAi technology, particularly with siRNA, is a powerful tool for gene silencing and therapeutic intervention.
Efficient in vivo delivery of RNAi therapeutics relies on advanced vector systems, primarily lipid-based.
Further development and characterization of delivery vehicles are essential for the clinical success of RNAi-based therapies.