Lumasiran (ALN-G01) 是一种 siRNA 化合物，通过靶向乙醇酸氧化酶 (glycolate oxidase) 减少肝脏草酸的产生。Lumasiran 通过沉默编码乙醇酸氧化酶的基因，消耗乙醇酸氧化酶，从而抑制草酸的合成，草酸是一种与原发性高草酸尿1型 (PH1) 表现直接相关的有毒代谢物。
货号:ajcx37500
CAS:1834610-13-7
分子式:N/A
分子量：16340.00
溶解度:N/A
纯度：95%
存储：Store at -20°C
库存：现货

Background:

Lumasiran (ALN-G01), a siRNA product, reduces hepatic oxalate production by targeting glycolate oxidase. By silencing the gene encoding glycolate oxidase, Lumasiran depletes glycolate oxidase and thereby inhibits the synthesis of oxalate, which is the toxic metabolite that is directly associated with the clinical manifestations of Primary hyperoxaluria type 1 (PH1)[1][2].

Lumasiran reduced urinary oxalate excretion, the cause of progressive kidney failure in PH1[1].

Lumasiran sodium is a subcutaneously administered, liver-directed RNA interference (RNAi) therapeutic agent[2].

[1]. Scott LJ, et al. Lumasiran: First Approval. Drugs. 2021;81(2):277-282.
[2]. Garrelfs SF, et al. Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1. N Engl J Med. 2021;384(13):1216-1226.