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In vivo evaluation of gene silencing with siRNAs showing the importance of an Rp isomer at the 5′ end of the antisense strand confirmed in rats. Female Sprague-Dawley rats (n = 3) were given a single, 2.5 mg/kg subcutaneous dose of indicated Ttr-targeted siRNA. Circulating TTR protein levels were measured by ELISA over a 50-day period post dose with each sample normalized to the pre-dose levels for the individual animal.
