CAS:1919820-28-2LTI-291

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LTI-291, a extract from patent WO 2017192841 A1, is an activator of glucocerebrosidase (Gcase) for treatment of Parkinson's disease.

规格	价格	促销价格	库存

18817579266

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18817579266

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实验数据

技术信息

CAS号

1919820-28-2

储存条件

分子式

C20H30N4O2

运输

蓝冰

分子量

358.48

别名

溶解度

DMSO	60.0 mg/mL (167.4 mM) warming
Water	insoluble

动物实验配方

生物活性

描述Mutations in GBA1, the gene encoding the lysosomal enzyme glucocerebrosidase, may lead to degradation of the protein, disruptions in lysosomal targeting and diminished performance of the enzyme in the lysosome, and are among the most common known genetic risk factors for the development of Parkinson disease and related synucleinopathies. Mutations in the gene encoding glucocerebrosidase (Gcase) are a risk factor for Parkinson's disease and diffuse Lewy Body Disease^[1]. LTI-291 is an activator of the GCase enzyme used for the treatment of Parkinson's disease. In a one-month phase 1b trial, there were no safety events and data showed a good dose-dependent brain penetration after 10 or 60 mg once daily oral treatment^[2].