| Description | NCGC 607 is a a noninhibitory chaperone of glucocerebrosidase (GCase). |
| In vitro | NCGC607是通过高通量筛选和化学结构优化确定的一种小分子非抑制性葡萄糖脑苷脂酶伴侣。该化合物成功地辅助了突变酶，恢复了葡萄糖脑苷脂酶的活性和蛋白水平，并在既定的iPSC衍生的巨噬细胞和多巴胺能神经元中减少了糖脂贮存，显示出其治疗神经病性高-糖病的潜力。此外，NCGC607在带有帕金森病表征的多巴胺能神经元中降低了α-突触核蛋白水平，暗示非抑制性小分子葡萄糖脑苷脂酶伴侣可作为帕金森病的潜在治疗方案。 |
| Synonyms | NCGC607 |
| molecular weight | 543.35 |
| Molecular formula | C24H22IN3O4 |
| CAS | 1462267-07-7 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice. |
| Solubility | DMSO: 55 mg/mL (101.22 mM) |
| References | 1. Aflaki E , Borger D K , Moaven N , et al. A New Glucocerebrosidase Chaperone Reduces α-Synuclein and Glycolipid Levels in iPSC-Derived Dopaminergic Neurons from Patients with Gaucher Disease and Parkinsonism[J]. Journal of Neuroscience the Official Journal of the Society for Neuroscience, 2016, 36(28):7441. |