Amelioration of a von Willebrand disease type 2B phenotype in vivo upon treatment with allele-selective siRNAs.

28 , 32 Although platelet counts were similar across treatment groups, we observed an expected trend toward fewer platelet aggregates after si Vwf.B6 treatment.

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Although the levels of B6 Vwf after both si Vwf and si Vwf.B6 treatment did not reach statistical significance compared with si Control.B6 treatment, the inhibition pattern observed was consistent with previous findings and displayed similarities to plasma VWF levels. 16 As expected, 129S Vwf mRNA levels did not differ between the siControl.B6- and si Vwf.B6 -treated 2B-B6.129S mice, further corroborating the allele selectivity of si Vwf.B6 in this VWD2B mouse model.

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