RNA interference (RNAi) may be used as a therapeutic for the bleeding disorder hemophilia. Specifically, artificially-generated, epigenetically-activated RNA molecules such as microRNAs can be used to inhibit gene expression through post-translational gene silencing. Researchers from France and the USA recently treated hemophiliac mice and macaques with a successful RNAi compound (ALN-AT3), which supresses the production of the anti-clotting substance antithrombin. Currently this therapeutic is being investigated in phase 1 clinical trials in individuals with hemophilia A or B.
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