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BioDrugs

Published in:

01-10-2009 | Review Article

RNA Interference Technologies and Therapeutics

From Basic Research to Products

Authors: Dr Marta López-Fraga, Tamara Martínez, Ana Jiménez

Published in: BioDrugs | Issue 5/2009

Abstract

RNA interference (RNAi) is a natural cellular process that regulates gene expression by a highly precise mechanism of sequence-directed gene silencing at the stage of translation by degrading specific messenger RNAs or blocking translation. In recent years, the use of RNAi for therapeutic applications has gained considerable momentum. It has been suggested that most of the novel disease-associated targets that have been identified are not ‘druggable’ with conventional approaches. However, any disease-causing gene and any cell type or tissue can potentially be targeted with RNAi.

This review focuses on the current knowledge of RNAi mechanisms and the safety issues associated with its potential use in a therapeutic setting. Some of the most important aspects to consider when working towards the application of RNAi-based products in a clinical setting have been related to achieving high efficacies and enhanced stability profiles through a careful design of the nucleic acid sequence and the introduction of chemical modifications, but most of all, to developing improved delivery systems, both viral and non-viral. These new delivery systems allow for these products to reach the desired target cells, tissues or organs in a highly specific manner and after administration of the lowest possible doses. Various routes of application and target locations are currently being addressed in order to develop effective delivery systems for different targets and pathologies, including infectious pathologies, genetic pathologies and diseases associated with dysregulation of endogenous microRNAs. As with any new technology, several challenges and important aspects to be considered have risen on the road to clinical intervention, e.g. correct design of preclinical toxicology studies, regulatory concerns, and intellectual property protection. The main advantages related to the use of RNAi-based products in a clinical setting, and the latest clinical and preclinical studies using these compounds, are reviewed.

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Title: RNA Interference Technologies and Therapeutics
From Basic Research to Products
Authors: Dr Marta López-Fraga
Tamara Martínez
Ana Jiménez
Publication date: 01-10-2009
Publisher: Springer International Publishing
Published in: BioDrugs / Issue 5/2009
Print ISSN: 1173-8804
Electronic ISSN: 1179-190X
DOI: https://doi.org/10.2165/11318190-000000000-00000

Keynote webinar | Spotlight on medication adherence

Springer Medicine

RNA Interference Technologies and Therapeutics

From Basic Research to Products

Abstract

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

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