Givosiran (ALN-AS1) 是一种小干扰 RNA，靶向肝脏氨基纤维素合酶 1 (ALAS1) 信使 RNA。Givosiran 下调 ALAS1 mRNA 并阻止神经毒性 δ-氨基纤维酸和卟啉的积累。Givosiran 可用于急性间歇性卟啉症的研究。
货号:ajcx35652
CAS:1639325-43-1
分子式:N/A
分子量：N/A
溶解度:N/A
纯度：98%
存储：Store at -20°C
库存：现货

Background:

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.