Patisiran sodium, a double-stranded small interfering RNA (siRNA), selectively targets a specific sequence within the transthyretin (TTR) messenger RNA (mRNA), effectively blocking hepatic synthesis of both mutant and wild-type TTR. It has significant applications in hereditary TTR amyloidosis research.

Patisiran sodium induces degradation of transthyretin (TTR) through RNA interference, leading to decreased serum TTR protein levels and a reduction in tissue TTR protein deposits. It achieves this by selectively targeting a conserved sequence in the 3' untranslated region of both mutant and wild-type TTR messenger RNA[2].