Method for enhancing mutant enzyme activity in gaucher disease
AssigneeMount Sinai School of Medicine
InventorsJian-Qiang Fan, Satoshi Ishii, Naoki Asano, Robert J. Desnick
Life of this patent
Estimating the term…
Abstract
Method for enhancing in a mammalian cell the activity of an enzyme associated with Gaucher Disease by administering a competitive inhibitor of glucocerebrosidase in an amount effective to enhance the activity of the enzyme. Preferred compounds for use in the method are imino sugars and related compounds. In particular, C8-12-alkyl derivatives of N-alkyl-deoxynojirimycin, isofagomine compounds, and calystegine compoiunds are effective to enhance glucocerebrosidase activity.
Claims34
A method of enhancing in a mammalian cell the activity of glucocerebrosidase, said method comprising administering a reversible competitive inhibitor of glucocerebrosidase in an amount effective to enhance the activity of glucocerebrosidase, and wherein said reversible competitive inhibitor is a specific competitive inhibitor for glucocerebrosidase.
A method of treating Gaucher disease comprising administering to an individual in need of treatment an effective amount of a reversible competitive inhibitor of glucocerebrosidase, and wherein said reversible competitive inhibitor is a specific competitive inhibitor for glucocerebrosidase.
Dependencies are inferred from the claim text.