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Givosiran

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Givosiran

Catalog No. T39123Cas No. 1639325-43-1

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.

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Product Introduction

Bioactivity

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

Chemical Properties

Cas No.

1639325-43-1

Storage & Solubility Information

Storage

Powder: -20°C for 3 years | In solvent: -80°C for 1 year

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