Powder: -20°C for 3 years | In solvent: -80°C for 1 year
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.
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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. 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. |
In vitro | Givosiran is conjugated to a trivalent N-acetylgalactosamine ligand for specific binding to the asialoglycoprotein receptor, facilitating targeted hepatocyte delivery[2]. |
Synonyms | ALN-AS1, Givosiran |
Molecular Weight | N/A |
CAS No. | 1639325-43-1 |
Powder: -20°C for 3 years | In solvent: -80°C for 1 year
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Givosiran 1639325-43-1 ALN-AS1 inhibitor inhibit