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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. By doing so, Givosiran effectively inhibits the accumulation of neurotoxic δ-aminolevulinic acid and porphobilinogen levels. This compound holds significant potential for further research on acute intermittent porphyria.
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. By doing so, Givosiran effectively inhibits the accumulation of neurotoxic δ-aminolevulinic acid and porphobilinogen levels. This compound holds significant potential for further research on acute intermittent porphyria.
规格 | 价格 | 库存 | 数量 |
---|---|---|---|
1 mg | 询价 | 期货 | |
5 mg | 询价 | 期货 |
产品描述 | 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. By doing so, Givosiran effectively inhibits the accumulation of neurotoxic δ-aminolevulinic acid and porphobilinogen levels. This compound holds significant potential for further research on acute intermittent porphyria. |
体外活性 | Givosiran is conjugated to a trivalent N-acetylgalactosamine ligand, which binds specifically to the asialoglycoprotein receptor, enabling targeted delivery to hepatocytes[2]. |
别名 | Givosiran, ALN-AS1 |
CAS No. | 1639325-43-1 |
存储 | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice. |
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