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.
Form:Solid
AI Insight
Names and Identifiers
Canonical SMILES
[Patisiran (sodium)]
Molecular Weight
13427.10 (AS: 6661.3; SS: 6765.8)
Certificates(CoA,COO,BSE/TSE and Analysis Chart)
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