Patisiran sodium_product_DataBase

Bioactivity	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].
Invitro	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].
Name	Patisiran sodium
CAS	1386913-72-9
Sequence	RNA, (A-U-G-G-A-A-Um-A-C-U-C-U-U-G-G-U-Um-A-C-dT-dT), complex with RNA (G-Um-A-ACm-Cm-A-A-G-A-G-Um-A-Um-Um-Cm-Cm-A-Um-dT-dT) (1:1) sodium salt
Molar Mass	13427.10 (AS: 6661.3; SS: 6765.8)
Appearance	Solid
Transport	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)
Reference	[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.

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