1639325-43-1

1639325-43-1结构式

常用中文名：Givosiran
常用英文名：Givosiran
CAS号：1639325-43-1
分子式：C₇₈H₁₃₉N₁₁O₃₀
分子量：1710.994
相关类别：研究领域神经疾病
发布时间：2022-01-23 15:39:46
更新时间：2025-08-27 18:00:19
Givosiran(ALN-AS1)是一种针对肝脏氨基乙酰丙酸合成酶1(ALAS1)信使RNA的小干扰RNA。Givosiran下调ALAS1 mRNA并防止神经毒性δ-氨基乙酰丙酸和疏孔素原水平的积累。Givosiran可用于急性间歇性卟啉症的研究[1][2]。

英文名	21-[(2-Acetamido-2-deoxy-β-D-galactopyranosyl)oxy]-N-[3-({5-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy]pentanoyl}amino)propyl]-6-[(3-{[3-({5-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy]pentanoyl}amino)propyl]amino}-3-oxopropoxy)methyl]-6-({12-[(2R,4R)-4-hydroxy-2-methyl-1-pyrrolidinyl]-12-oxododecanoyl}amino)-11,17-dioxo-4,8-dioxa-12,16-diazahenicosan-1-amide
英文别名	21-[(2-Acetamido-2-deoxy-β-D-galactopyranosyl)oxy]-N-[3-({5-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy]pentanoyl}amino)propyl]-6-[(3-{[3-({5-[(2-acetamido-2-deoxy-β-D-galactopyranosyl)oxy ]pentanoyl}amino)propyl]amino}-3-oxopropoxy)methyl]-6-({12-[(2R,4R)-4-hydroxy-2-methyl-1-pyrrolidinyl]-12-oxododecanoyl}amino)-11,17-dioxo-4,8-dioxa-12,16-diazahenicosan-1-amide 1-Pyrrolidinedodecanamide, N-[2-[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]propyl]amino]-3-oxopropoxy]-1,1-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-D-gala ctopyranosyl]oxy]-1-oxopentyl]amino]propyl]amino]-3-oxopropoxy]methyl]ethyl]-4-hydroxy-2-methyl-λ-oxo-, (2R,4R)-

描述	Givosiran(ALN-AS1)是一种针对肝脏氨基乙酰丙酸合成酶1(ALAS1)信使RNA的小干扰RNA。Givosiran下调ALAS1 mRNA并防止神经毒性δ-氨基乙酰丙酸和疏孔素原水平的积累。Givosiran可用于急性间歇性卟啉症的研究[1][2]。
相关类别	信号通路 >> 其他 >> 其他研究领域 >> 神经疾病
体外研究	Givosiran与三价N-乙酰半乳糖胺配体结合,该配体与去唾液酸糖蛋白受体特异性结合,从而实现向肝细胞的靶向递送[2]。
参考文献	[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.