Givosiran (Synonyms: ALN-AS1) |
| Katalog-Nr.GC63424 |
Givosiran (ALN-AS1) ist eine kleine interferierende RNA, die auf die messenger RNA der hepatischen Aminolävulinsynthase 1 (ALAS1) abzielt.
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Cas No.: 1639325-43-1
Sample solution is provided at 25 µL, 10mM.
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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 and porphobilinogen levels. Givosiran can be used for the research of acute intermittent porphyria[1][2].
Givosiran is conjugated to a trivalent N-acetylgalactosamine ligand, which binds specifically to the asialoglycoprotein receptor, enabling targeted delivery to hepatocytes[2].
[1]. Scott LJ. Givosiran: First Approval. Drugs. 2020 Feb;80(3):335-339.
[2]. Balwani M, et, al. Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria. N Engl J Med. 2020 Jun 11;382(24):2289-2301.
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