About: RNA Interference Technologies and Therapeutics: From Basic Research to Products

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An Entity of Type : schema:ScholarlyArticle, within Data Space : wasabi.inria.fr associated with source document(s)

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type	Academic Article research paper schema:ScholarlyArticle
isDefinedBy	Covid-on-the-Web dataset
title	RNA Interference Technologies and Therapeutics: From Basic Research to Products
Creator	Jiménez, Ana López-Fraga, Marta Martínez, Tamara
source	Medline; PMC
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.
has issue date	2012-08-16(xsd:dateTime)
bibo:doi	10.2165/11318190-000000000-00000
bibo:pmid	19754220
has license	no-cc
sha1sum (hex)	478dd98749291f192b9ce6b8e68c5acf909415b3
schema:url	https://doi.org/10.2165/11318190-000000000-00000
resource representing a document's title	RNA Interference Technologies and Therapeutics: From Basic Research to Products
has PubMed Central identifier	PMC7099360
has PubMed identifier	19754220
schema:publication	BioDrugs
resource representing a document's body	covid:478dd98749291f192b9ce6b8e68c5acf909415b3#body_text
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