acid β-glucosidase

Afegostat is a pharmacological chaperone that specifically and reversibly binds acid-β-glucosidase (GCase) in the endoplasmic reticulum with high affinity.

2'-Hydroxygenistein是一种天然的黄酮，具有抗氧化、抗炎、抗癌、抗子宫内膜增生等多种潜力。2'-羟基金雀异黄素是多种酶抑制剂，包括吲哚-3-乙酸氧化酶、α-葡萄糖苷酶、β-葡萄糖醛酸酶和溶菌酶。

Glucosylsphingosine(Lyso-Gb1) 是一种天然存在的糖基神经酰胺, 是GBA (acid β-glucosidase）的代谢产物, 参与细胞识别、信号转导和细胞间相互作用。Glucopsychosine在某些神经退行性疾病（如 Gaucher病）中积累，通过激活哺乳动物雷帕霉素复合体1（mTORC1）来破坏溶酶体功能,也是一种非竞争性糖脑苷酶 (glucocerebrosidase) 抑制剂。

Afegostat is an acid β-glucosidase (GCase) active site inhibitor that acts as a pharmacological chaperone. Afegostat inhibits GCase with K(i) ~30 nM for wild-type and mutant enzymes. Afegostat was an experimental drug for the treatment of certain forms of

Afegostat D-Tartrate is a pharmacological chaperone that specifically and reversibly binds acid-β-glucosidase (GCase) in the endoplasmic reticulum with high affinity.

Afegostat, aslo known as Isofagomine, and AT2101, was an experimental drug for the treatment of certain forms of Gaucher's disease. Isofagomine (IFG) is an acid β-glucosidase (GCase) active site inhibitor that acts as a pharmacological chaperone. FG inhibits GCase with K(i) ~30 nM for wild-type and mutant enzymes (N370S and V394L). Administration of IFG (30 mg kg day) to the mice homozygous for GCase mutations (V394L, D409H, or D409V) led to increased GCase activity in visceral tissues and brain extracts.