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Figure 7 | Silence

Figure 7

From: In vivo quantification of formulated and chemically modified small interfering RNA by heating-in-Triton quantitative reverse transcription polymerase chain reaction (HIT qRT-PCR)

Figure 7

Correlation between in vivo delivered small interfering RNA (siRNA) and target knockdown in rat liver. (a) Experimental setup. Rats were tail-vein injected with phosphate-buffered saline (PBS), 0.0625, 0.125 and 0.25 mg/kg chemically modified and formulated siRNA in lipid nanoparticle (LNP)-KC2 (LNP-KC2-AD1661) on day 0. At 1, 3, 7, 10 and 20 days after injection, frozen powdered liver from three rats from each group was analysed for siRNA concentration and for target (factor VII (FVII)) expression. (b) Quantification of modified siRNA in rat liver. Average of three rats, measured twice independently, with day 20 levels below lower limit detection and therefore not shown (lowest limit of detection (PBS) = 25.8 ± 4.6 pg/g). (c) Gene target knockdown in rat liver. The average of FVII RNA expression in three rats, relative to FVII expression in PBS injected rats. (d) Correlation between siRNA drug levels and FVII RNA levels over time in rat livers. Symbols represent means ± 1 standard error (SE) for individual rats. Blue line, linear regression; grey shading indicates 95% confidence interval. Yellow rectangles indicate 50% effective dose (EC50) and EC80 estimates.

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ISSN: 1758-907X