Patisiran sodium is a double-stranded small interfering RNA that targets a sequence within the transthyretin (TTR) messenger RNA. Patisiran sodium specifically inhibits hepatic synthesis of mutant and wild-type TTR. Patisiran sodium can be used for the research of hereditary TTR amyloidosis .
In Vitro
Patisiran sodium causes TTR degradation (via RNA interference) and subsequently a reduction in serum TTR protein levels and tissue TTR protein deposits by specifically binding to a genetically conserved sequence in the 3' untranslated region of mutant and wild-type transthyretin (TTR) messenger RNA. MCE has not independently confirmed the accuracy of these methods. They are for reference only.
