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Knockdown stands up

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Abstract

In the past year, the genetic research of mammalian cells in vitro has gained the advantages of RNA interference (RNAi), a process found in worms and plants by which double stranded RNAs mediate selective gene inactivation through mRNA destruction. Recently, two papers have shown that genes could be suppressed in vivo in mammals by RNAi, which has potential implications for both therapeutics and research.

Section snippets

RNAi in mammals

The discovery of RNA interference (RNAi) whereby double stranded (ds) RNA molecules suppress the expression of complementary genes has endowed research with a whole new set of tools that facilitate genetic studies of somatic cells and could possibly be applied in the clinic. RNAi has been known for some years now in organisms such as C. elegans and plants 1, 2. However, a much wider interest in this phenomenon was sparked by the discovery that dsRNA can selectively suppress gene expression in

In vivo

Introduction of long dsRNAs into worms and plants, either by transgenes, injections, or feeding results in RNAi that is observed throughout almost the whole organism. However, until recently there have been no reports of in vivo use of RNAi in mammals. This gap has now been filled by two recent reports that show proofs of principle for the use of RNAi in vivo in mammals 8, 9. In the paper by McCaffrey et al., synthetic siRNAs or a vector coding for siRNA against luciferase were co-transfected

New tools for therapy and research

A prerequisite for efficient and stable delivery of siRNA expression in cells would be the incorporation of its expression cassette in viral vectors. This is not a trivial condition because the major function of RNAi in plants and worms, and perhaps in mammals, is to suppress viral expression itself. However, using a self-inactivating siRNA-expressing retrovirus, we recently showed that such an approach is feasible [15]. We used the high sequence-selectivity of the siRNA system to target only

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    RNAi involves the post-transcriptional silencing of genes through the formation of dsRNA segments within mRNA transcripts, resulting in the degradation of the corresponding mRNA [5]. While RNAi is rapidly becoming a powerful tool for gene silencing, the precise mechanism whereby RNAi silences genes is not completely understood [6,7]. RNAi is a naturally occurring process that leads to the 'silencing' of genes.

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