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编 号：F197549
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CAS No: 1639325-43-1
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生物活性：
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.
体外研究：
Givosiran 与三价 N-乙酰半乳糖胺配体偶联，后者特异性结合脱唾液酸糖蛋白受体，从而能够靶向递送至肝细胞。
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.
体外研究：
Givosiran 与三价 N-乙酰半乳糖胺配体偶联，后者特异性结合脱唾液酸糖蛋白受体，从而能够靶向递送至肝细胞。
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