gcase

Glucocerebrosidase (Glucosylceramidase; GBA), a lysosomal enzyme, catalyzes the hydrolysis of the β-glucosidic linkage in glucocerebroside (GC) to yield glucose and ceramide [1].

Gcase activator 2 is a β-glucocerebrosidase activator that induces dimerization of GCase at the endoplasmic reticulum (ER) and lysosomal pHs, increases lysosomal substrate metabolism, and has therapeutic potential for the treatment of Parkinson's and Gosheimer's diseases.

Gcase activator 3 is a glucocerebrosidase (GCase) activator, promoting increased GCase activity in GBA1 mutant fibroblasts. Gcase activator 3 is useful for studying Parkinson's disease.

GCase Modulator-1 (Compound 9g), a quinazoline derivative, functions as a glucosidase modulator with an AC50 value of 2.23 µM [1].

Gcase activator 1 (LTI-291) is a glucocerebrosidase (Gcase) activator.

Endoglycoceramidase II (EGCase II), an endo-β-glucosidase, catalyzes the hydrolysis of the β-glycosidic linkage between oligosaccharides and ceramides in glycosphingolipids, releasing the complete glycan from ceramide [1] [2].

Endoglycoceramidase I (EGCase I), a glycosidase frequently utilized in biochemical studies, catalyzes a transglycosylation reaction. This process involves transferring the sugar moiety of GSLs to the primary hydroxyl group of various 1-alkanols [1].

PFB-FDGlu is a lysosomal Glucocerebrosidase (GCase) substrate with cell-permeable specificity that is cleaved to produce fluorescein.PFB-FDGlu is often used in conjunction with a flow cytometer to measure GCase activity in living cells on an individual cell basis.

Conduritol B epoxide is an irreversible inhibitor of β-glucosidase (GCase).

4-Methylumbelliferyl-β-D-Glucopyranoside (4-MU-GLU) is used in the GCase activity assay based on the catalytic hydrolysis of 4-methylumbelliferyl β-D-glucopyranoside that releases the highly fluorescent 4-methylumbelliferyl (4-MU).

Ambroxol (NA-872) is a secretolytic agent used in the treatment of respiratory diseases associated with viscid or excessive mucus. It is the active ingredient of Mucosolvan, Lasolvan or Mucoangin. The substance is a mucoactive drug with several properties including secretolytic and secretomotoric actions that restore the physiological clearance mechanisms of the respiratory tract which play an important role in the body's natural defense mechanisms. It stimulates synthesis and release of surfactant by type II pneumocytes. Surfactants act as an anti-glue factor by reducing the adhesion of mucus to the bronchial wall, in improving its transport and in providing protection against infection and irritating agents.

Ambroxol hydrochloride (Mucosolvan) is a metabolite of BROMHEXINE that stimulates mucociliary action and clears the air passages in the respiratory tract. It is usually administered as the hydrochloride.

ML198 is a novel activator and non-inhibitory chaperone of glucocerebrosidase. ML198 serves as a starting point to develop a novel approach towards small molecule treatment for patients suffering from Gaucher disease.

NCGC 607 is a a noninhibitory chaperone of glucocerebrosidase (GCase).

ML266, a glucocerebrosidase (GCase) chaperone with an IC50 of 2.5 µM, facilitates the transport of the mutant protein to the lysosome, thereby restoring GCase activity without inhibiting the enzyme's function. This compound shows promise for Gaucher disease research.

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

Glucocerebrosidase-IN-1 (compound 11a) hydrochloride is a potent and selective glucocerebrosidase (GCase) inhibitor with IC50 of 29.3 μM and Ki of 18.5 μM, used in the research of Gaucher disease (GD) and Parkinson's disease (PD) [1].

Glucocerebrosidase-IN-1 (compound 11a) is a potent and selective GCase (glucocerebrosidase) inhibitor, with an IC50 of 29.3 μM and a Ki of 18.5 μM. Due to GCase involvement in pathological disorders consequent to enzyme deficiency, Glucocerebrosidase-IN-1 can be used for research of Gaucher disease (GD) and Parkinson's disease (PD)[1].

Ambroxol acefylline, an active metabolite of the proagent Bromhexine, has potent expectorant effects and functions as a glucocerebrosidase (GCase) chaperone, increasing GCase activity. It induces lung autophagy and has potential applications in Parkinson's disease and neuronopathic Gaucher disease research [1][2].

AMP-Deoxynojirimycin (AMP-DNM) is a potent inhibitor of ceramide glucosyltransferase and GCase 2, as well as GlcCer biosynthesis, and is utilized in the study of Parkinson's disease and diabetes.

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

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, 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.