gcase

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.

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

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.

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.

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].

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].

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.

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.

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

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

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

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.

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

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

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

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

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.

Glucocerebrosidase-IN-1 (compound 11a) is a potent and selective GCase (glucocerebrosidase) inhibitor, with an IC 50 of 29.3 μM and a K i of 18.5 μM. Due to GCase involvement in pathological disorders consequent to enzyme deficiency, Glucocerebrosidase-IN-1 can be used for the research of Gaucher disease (GD) and Parkinson’s disease (PD)[1].