AS-Patisiran (sodium)

AS-Patisiran sodium is an antisense strand of Patisiran. Patisiran is a double-stranded small interfering RNA that targets a sequence within the transthyretin (TTR) messenger RNA. Patisiran specifically inhibits hepatic synthesis of mutant and wild-type TTR. Patisiran can be used for the research of hereditary TTR amyloidosis[1][2][3].

Product Specifications

UNSPSC

12352208

Target

Small Interfering RNA (siRNA) ; Transthyretin (TTR)

Type

Oligonucleotides

Related Pathways

Epigenetics; Neuronal Signaling

Applications

Neuroscience-Neuromodulation

Field of Research

Neurological Disease

Assay Protocol

https://www.medchemexpress.com/as-patisiran.html

Purity

95.10

Solubility

H2O : ≥ 100 mg/mL

Smiles

[AS-Patisiran(sodium)]

Molecular Weight

6661.30

References & Citations

[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.