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编 号:F747473
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CAS No: 1834610-13-7
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产品详情
生物活性:
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).
体内研究:
Lumasiran 是一种皮下给药的肝脏定向 RNA 干扰 (RNAi) 抑制剂。
体外研究:
Lumasiran 减少尿草酸盐排泄,这是 PH1 进行性肾衰竭的原因。