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Towards in vivo application of RNA interference – new toys, old problems

Sascha Rutz and Alexander Scheffold*

Author Affiliations

Deutsches Rheuma-Forschungszentrum Berlin, Berlin, Germany

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Arthritis Res Ther 2004, 6:78-85  doi:10.1186/ar1168

Published: 10 March 2004


RNA interference (RNAi) is the sequence-specific degradation of mRNA by short double-stranded RNA molecules. The technology, introduced only 5 years ago, has stimulated many fantasies regarding the future of functional gene analysis and gene therapy. Given its ease of application, its high efficiency and remarkable specificity, RNAi holds great promise for broad in vitro and in vivo application in all areas of biomedicine. Despite its potential, the major obstacle to the use of RNAi (as for all previous gene silencing approaches) is the need for efficient and sustained delivery of small interfering RNA into primary mammalian cells, and specific targeting of particular cell types in vivo.

functional genomics; gene silencing; primary mammalian cell; small interfering RNA; transfection