Trends in Biotechnology
Research FocusKnockdown stands up
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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Cited by (34)
RNA interference and CRISPR/Cas9 techniques for controlling mycotoxins
2021, CRISPR and RNAi Systems: Nanobiotechnology Approaches to Plant Breeding and Protection: A Volume in Nanobiotechnology for Plant ProtectionRNA interference technology used for the study of aquatic virus infections
2014, Fish and Shellfish ImmunologyCitation Excerpt :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.
RNA interference: A futuristic tool and its therapeutic applications
2012, Saudi Journal of Biological SciencesCitation Excerpt :Studying the effects of this decrease can show the physiological role of the gene product. Since RNAi may not totally abolish expression of the gene, this technique is sometimes referred as a “knockdown”, to distinguish it from “knockout” procedures in which expression of a gene is entirely eliminated (Voorhoeve and Agami, 2003). Extensive efforts in computational biology have been directed toward the design of successful dsRNA reagents that maximize gene knockdown but minimize “off-target” effects.
Developments in effective application of small inhibitory RNA (siRNA) technology in mammalian cells
2004, Drug Discovery Today: TARGETSSilencing structural and nonstructural genes in baculovirus by RNA interference
2004, Virus ResearchGenetic modification of the heart: Exploring necessity and sufficiency in the past 10 years
2004, Journal of Molecular and Cellular Cardiology