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PubMed · 14723975

Therapeutic siRNAs.

Mouldy Sioud
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Abstract

The ability of small-interfering RNAs (siRNAs) to silence gene expression in somatic mammalian cells has provided researchers with a novel tool to block the expression of disease-causing genes, provided that their mRNA sequences are known. siRNAs can be delivered to cells either exogenously as synthetic agents or endogenously as gene-encoding siRNAs. Recent studies demonstrate the general application of siRNAs to silence gene expression in a range of cell types and in whole mammals. Beyond their value for dissecting gene functions and target validation, siRNAs also hold great potential as gene-specific therapeutic agents.

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AnimalsCells, CulturedDisease Models, AnimalGene Expression Regulation, NeoplasticHumansMolecular BiologyRNA, Small InterferingVirus DiseasesJournal ArticleResearch Support, Non-U.S. Gov'tReview

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Mouldy Sioud. 2004. Therapeutic siRNAs.. https://doi.org/10.1016/j.tips.2003.11.006

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