Answer
Patisiran sodium (Onpattro) delivers small interfering RNA to liver cells in lipid nanoparticles, triggering RNA interference that degrades variant and wild-type transthyretin messenger RNA and thereby reduces serum transthyretin protein.
Sources for this answer
- Onpattro 2 mg/mL concentrate for solution for infusion.Alnylam UK Ltd · emc · SmPC §5.1 Pharmacodynamic properties · Version 12Document updated
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