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.
In Vitro
Givosiran is conjugated to a trivalent N-acetylgalactosamine ligand, which binds specifically to the asialoglycoprotein receptor, enabling targeted delivery to hepatocytes. MCE has not independently confirmed the accuracy of these methods. They are for reference only.
Form:Solid
规格
别名
吉沃西兰
规格或纯度
≥97%
英文名称
Givosiran
生化机理
Givosiran(ALN-AS1)是一种靶向肝脏氨基乙酰丙酸合成酶 1(ALAS1)信使 RNA 的小干扰 RNA。Givosiran 可下调 ALAS1 mRNA,防止神经毒性δ-氨基乙酰丙酸和卟啉原水平的积累。Givosiran c