[CAS NO. 1386913-72-9] Patisiransodium
PRODUCTS SPECIFICATIONS [1386913-72-9]
DESCRIPTION [1386913-72-9]
Overview
| MDL | - |
|---|---|
| Molecular Weight | 13427.10 (AS: 6661.3; SS: 6765.8) |
| Molecular Formula | - |
| SMILES | [Patisiran (sodium)] |
Summary
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 [1] [2] [3] .
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] .
MCE has not independently confirmed the accuracy of these methods. They are for reference only.
Appearance
Solid
Shipping
Room temperature in continental US; may vary elsewhere.
Storage
-20°C, stored under nitrogen
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen)
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.