Givosiran (Synonyms: ALN-AS1) |
カタログ番号GC63424 |
ギボシラン(ALN-AS1)は、肝臓アミノレブリン酸合成酵素1(ALAS1)メッセンジャーRNAを標的とする小さな干渉RNAです。
Products are for research use only. Not for human use. We do not sell to patients.
Cas No.: 1639325-43-1
Sample solution is provided at 25 µL, 10mM.
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.
Average Rating: 5
(Based on Reviews and 29 reference(s) in Google Scholar.)GLPBIO products are for RESEARCH USE ONLY. Please make sure your review or question is research based.
Required fields are marked with *