产品说明书
Gcase activator 1
 |
同义名 : | LTI-291 |
| CAS号 : | 1919820-28-2 | |
| 货号 : | A181720 | |
| 分子式 : | C20H30N4O2 | |
| 纯度 : | 99%+ | |
| 分子量 : | 358.478 | |
| MDL号 : | MFCD31618016 | |
| 存储条件： |
粉末 Inert atmosphere,Room Temperature 液体 -20°C:3-6个月-80°C:12个月 |
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| 溶解度 : |
DMSO: 50 mg/mL(139.48 mM)，配合低频超声助溶，注意：DMSO长时间开封后，会吸水并导致溶解能力下降，请避免使用长期开封的DMSO |
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| 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]. | ||
| 实验方案 | |||
|---|---|---|---|
| 1mg | 5mg | 10mg | |
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1 mM 5 mM 10 mM |
2.79mL 0.56mL 0.28mL |
13.95mL 2.79mL 1.39mL |
27.90mL 5.58mL 2.79mL |
| 参考文献 |
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