| Description | Patisiran sodium is a double-stranded small interfering RNA (siRNA) that selectively targets a specific sequence within the transthyretin (TTR) messenger RNA (mRNA). By doing so, it effectively blocks the hepatic synthesis of both mutant and wild-type TTR. Patisiran sodium has significant applications in the field of hereditary TTR amyloidosis research. |
| 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[2]. |
| molecular weight | N/A |
| CAS | 1386913-72-9 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year |
| References | 1. Adams D, et, al. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018 Jul 5;379(1):11-21. 2. Hoy SM. Patisiran: First Global Approval. Drugs. 2018 Oct;78(15):1625-1631. 3. Kristen AV, et, al. Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis. Neurodegener Dis Manag. 2019 Feb;9(1):5-23. |