Bioactivity | Afegostat is a pharmacological chaperone, which specifically and reversibly binds acid-β-glucosidase (GCase) in the endoplasmic reticulum (ER) with high affinity[1]. |
Target | GCase |
In Vivo | Afegostat (AT2101) increases GCase activity in brain, liver, and spleen. Afegostat (100 mg/kg) administrates orally for 4 months to Thy1-aSyn mice improved motor and nonmotor function, abolishes microglial inflammatory response in the substantia nigra, reduces α-synuclein immunoreactivity in nigral dopaminergic neurons, and reduces the number of small α-synuclein aggregates, while increasing the number of large α-synuclein aggregates[1]. |
Name | Afegostat |
CAS | 169105-89-9 |
Formula | C6H13NO3 |
Molar Mass | 147.17 |
Transport | Room temperature in continental US; may vary elsewhere. |
Storage | Please store the product under the recommended conditions in the Certificate of Analysis. |
Reference | [1]. Richter F, et al. A GCase chaperone improves motor function in a mouse model of synucleinopathy. Neurotherapeutics. 2014 Oct;11(4):840-56. |