1639325-43-1
1639325-43-1
Chemical Structure
Givosiran
Synonym(s): ALN-AS1
- CAS No.: 1639325-43-1
- Formula:N/A
- Molecular Weight:16300.60
SMILES: [Givosiran]
Biological Activity: Givosiran (ALN-AS1) is a small interfering RNA that targets hepatic aminolevulinate synthase 1 (ALAS1) messenger RNA. Givosiran downregulates ALAS1 mRNA and prevents accumulation of neurotoxic δ-aminolevulinic acid (ALA) and porphobilinogen (PBG) levels. Givosiran demonstrates potent inhibitory activity against ALAS1 in mouse, rat, and cynomolgus monkey models. Givosiran can be used for the research of acute hepatic porphyria (AHP)[1][2].
| Cat. No. | Product Name | Purity | Description | Pricing | |||||||||||||||||||
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Givosiran | 94.21% | Givosiran (ALN-AS1) is a small interfering RNA that targets hepatic aminolevulinate synthase 1 (ALAS1) messenger RNA. Givosiran downregulates ALAS1 mRNA and prevents accumulation of neurotoxic δ-aminolevulinic acid (ALA) and porphobilinogen (PBG) levels. Givosiran demonstrates potent inhibitory activity against ALAS1 in mouse, rat, and cynomolgus monkey models. Givosiran can be used for the research of acute hepatic porphyria (AHP). | ||||||||||||||||||||
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References
- [1]. Scott LJ. Givosiran: First Approval. Drugs. 2020 Feb;80(3):335-339. [Content Brief]
- [2]. Chan A, et al. Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification. Mol Ther Nucleic Acids. 2015 Nov 3;4(11):e263. [Content Brief]
Keywords
