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Experimental RNAi

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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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Updated: Jul 15, 2026

Studying Protein Function and the Role of Altered Protein Expression by Antibody Interference and Three-dimensional Reconstructions

Studying Protein Function and the Role of Altered Protein Expression by Antibody Interference and Three-dimensional Reconstructions

Published on: April 21, 2016

RNAi therapeutics: principles, prospects and challenges.

Lars Aagaard¹, John J Rossi

¹Division of Molecular Biology, Beckman Research Institute of City of Hope, Duarte, CA 91010, USA.

Advanced Drug Delivery Reviews

|April 24, 2007

Summary

RNA interference (RNAi) offers potent gene silencing for therapeutics. Overcoming challenges like off-target effects and delivery is key to realizing RNAi

Area of Science:

Biotechnology
Molecular Biology
Genetics

Background:

RNA interference (RNAi) is a recently discovered biological process with therapeutic potential.
It leverages cellular machinery for highly effective gene expression down-regulation.
RNAi-based therapies are advancing, with human clinical trials underway.

Purpose of the Study:

To review the mechanistic aspects of RNA interference.
To discuss challenges and potential solutions for therapeutic RNAi applications.
To explore the future therapeutic applications of RNAi.

Main Methods:

Review of existing literature on RNA interference mechanisms.
Analysis of challenges including off-target effects and immune responses.

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Discussion of in vivo delivery strategies and therapeutic potential.

Main Results:

RNAi demonstrates potent gene silencing capabilities.
Key challenges include off-target effects, type I interferon responses, and in vivo delivery.
Solutions and strategies to mitigate these hurdles are being developed.

Conclusions:

RNA interference holds significant promise as a major therapeutic modality.
Further investigation into RNAi mechanisms is warranted.
Addressing current challenges will facilitate the clinical translation of RNAi therapies.