Nature Biotechnology 23, 1399 - 1405 (2005)
Published online: 4 November 2005; | doi:10.1038/nbt1161
Activation of the mammalian immune system by siRNAsJoao T Marques
& Bryan R G Williams
Department of Cancer Biology, Lerner Research Institute, Cleveland Clinic Foundation, 9500 Euclid Avenue, Cleveland, Ohio, 44195, USA.
Correspondence should be addressed to Bryan R G Williams williab@ccf.org Inhibition of gene expression through RNA interference (RNAi) is emerging as a powerful experimental tool for gene function and target validation studies. The potential uses of this technology seem unlimited, extending to the prevention and therapy of human diseases. However, recent work demonstrating that there are unanticipated, different nonspecific effects associated with the use of small interfering RNAs in mammals has raised concerns about the safe use of RNAi in vivo. These nonspecific effects include activation of the immune system, potentially harming the individual. The application of screening assays for nonspecific activation of both innate and acquired immunity will be necessary for further development of RNAi as a therapeutic tool.
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