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Mitapivat

CAS No. : 1260075-17-9

MCE 國際站：Mitapivat

產(chǎn)品活性：Mitapivat (AG-348) 是一種口服有效的丙酮酸激酶 (pyruvate kinase) 變構激活劑。Mitapivat 在廣泛的 PKLR 基因型中增加酶活性、蛋白質穩(wěn)定性和 ATP 水平，具有恢復丙酮酸激酶缺乏的糖酵解途徑活性的潛力。Mitapivat 可用于丙酮酸激酶缺乏癥的研究。

研究領域：Metabolic Enzyme/Protease

作用靶點：Pyruvate Kinase

In Vitro: Mitapivat (0.1 nM-100 μM; 16 h) activates WT PK-R in RBCs from healthy donors.
Mitapivat (0.01 nM-10 μM; 16 h) promotes production of ATP in RBC cells in a dose-dependent manner.

In Vivo: Mitapivat (50 mg/kg; p.o.; twice daily for 21 days) improves anemia in a mouse model for β-thalassemia.

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