Method for increasing the activity of lysosomal enzymes
View Patent ↗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 compounds are effective to enhance glucocerebrosidase activity.
1. Method of treating Hurler-Scheie disease in a subject in need thereof, comprising administering to the subject an effective amount of a competitive inhibitor of α-L-iduronidase.
2. The method of claim 1 , wherein the competitive inhibitor is 1-deoxyiduronojirimycin.
3. The method of claim 1 , wherein the competitive inhibitor is 2-carboxy-3,4,5-trideoxypiperidine.
4. A method for increasing in a mammalian cell the activity of α-L-iduronidase, which method comprises contacting the cell with a reversible competitive inhibitor of the α-L-iduronidase in an amount effective to increase α-L-iduronidase activity.
5. The method of claim 4 , wherein the competitive inhibitor is 1-deoxyiduronojirimycin.
6. The method of claim 4 , wherein the competitive inhibitor is 2-carboxy-3,4,5-trideoxypiperidine.