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Pack Size | Price | Availability | Quantity |
---|---|---|---|
1 mg | Inquiry | Backorder | |
5 mg | Inquiry | Backorder |
Description | Givosiran (ALN-AS1) is a small interfering RNA (siRNA) compound that specifically targets hepatic aminolevulinate synthase 1 (ALAS1) messenger RNA (mRNA) to downregulate its expression, thereby inhibiting the accumulation of neurotoxic δ-aminolevulinic acid and porphobilinogen levels, with significant potential for further research on acute intermittent porphyria. |
In vitro | Givosiran is conjugated to a trivalent N-acetylgalactosamine ligand for specific binding to the asialoglycoprotein receptor, facilitating targeted hepatocyte delivery[2]. |
Alias | ALN-AS1, Givosiran |
Cas No. | 1639325-43-1 |
Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year |
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