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Givosiran

CAT: 0804-HY-132610-01Size: 1 mgDry Ice: NoHazardous: No
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CAT#:0804-HY-132610-01Size:1 mg
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24/48H Stock Items & 2 to 6 Weeks non Stock Items.
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Description
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].
CAS Number
1639325-43-1
Product Name Alternative
ALN-AS1
UNSPSC
12352208
Target
Small Interfering RNA (siRNA)
Type
Oligonucleotides
Related Pathways
Epigenetics
Applications
Neuroscience-Neuromodulation
Field of Research
Metabolic Disease
Assay Protocol
https://www.medchemexpress.com/givosiran.html
Purity
98.51
Solubility
10 mM in H2O
Smiles
[Givosiran]
Molecular Formula
C524H694F16N173O316P43S6
Molecular Weight
16300.60
References & Citations
[1]Scott LJ. Givosiran: First Approval. Drugs. 2020 Feb;80 (3) :335-339.|[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.
Shipping Conditions
Blue Ice
Storage Conditions
-20°C (Powder, sealed storage, away from moisture)
Scientific Category
Oligonucleotides
Clinical Information
Launched

Alternative Products

CatalogName
T39123-01Givosiran

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